{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,15]],"date-time":"2026-09-15T23:18:04Z","timestamp":1789514284965,"version":"build-2803163510"},"reference-count":33,"publisher":"Massachusetts Medical Society","issue":"25","license":[{"start":{"date-parts":[[2022,12,22]],"date-time":"2022-12-22T00:00:00Z","timestamp":1671667200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/www.nejmgroup.org\/legal\/terms-of-use.htm"}],"funder":[{"DOI":"10.13039\/100000900","name":"California Institute for Regenerative Medicine","doi-asserted-by":"publisher","award":["CLIN2-10830"],"award-info":[{"award-number":["CLIN2-10830"]}],"id":[{"id":"10.13039\/100000900","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000060","name":"National Institute of Allergy and Infectious Diseases","doi-asserted-by":"publisher","award":["U54-AI082973"],"award-info":[{"award-number":["U54-AI082973"]}],"id":[{"id":"10.13039\/100000060","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["N Engl J Med"],"published-print":{"date-parts":[[2022,12,22]]},"DOI":"10.1056\/nejmoa2206575","type":"journal-article","created":{"date-parts":[[2022,12,21]],"date-time":"2022-12-21T22:01:06Z","timestamp":1671660066000},"page":"2344-2355","source":"Crossref","is-referenced-by-count":79,"title":["Lentiviral Gene Therapy for Artemis-Deficient SCID"],"prefix":"10.1056","volume":"387","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1088-8045","authenticated-orcid":false,"given":"Morton J.","family":"Cowan","sequence":"first","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jason","family":"Yu","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janelle","family":"Facchino","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carol","family":"Fraser-Browne","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ukina","family":"Sanford","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Misako","family":"Kawahara","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jasmeen","family":"Dara","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janel","family":"Long-Boyle","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jess","family":"Oh","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wendy","family":"Chan","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shivali","family":"Chag","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lori","family":"Broderick","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Deepak","family":"Chellapandian","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H\u00e9l\u00e8ne","family":"Decaluwe","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine","family":"Golski","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diana","family":"Hu","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline Y.","family":"Kuo","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Holly K.","family":"Miller","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aleksandra","family":"Petrovic","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Currier","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joan F.","family":"Hilton","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Divya","family":"Punwani","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6146-3952","authenticated-orcid":false,"given":"Christopher C.","family":"Dvorak","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Harry L.","family":"Malech","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R. Scott","family":"McIvor","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6623-6276","authenticated-orcid":false,"given":"Jennifer M.","family":"Puck","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics (M.J.C., J.Y., J.F., C.F.-B., U.S., M.K., J.D., J.L.-B., W.C., S.C., R.C., C.C.D., J.M.P.) and Epidemiology and Biostatistics (J.F.H.), the Smith Cardiovascular Research Institute (M.J.C., J.M.P.), and the School of Pharmacy (J.L.-B.), University of California, San Francisco (UCSF), and UCSF Benioff Children\u2019s Hospital (M.J.C., J.F., J.D., J.L.-B., J.O., C.C.D., J.M.P.), San Francisco, the Department of Pediatrics, University of California, San Diego, and Rady Children\u2019s Hospital, San Diego (L.B.), and the Department of Pediatrics, UCLA Mattel Children\u2019s Hospital, Los Angeles (C.Y.K.) \u2014 all in California; the Department of Pediatrics, Johns Hopkins All Children\u2019s Hospital, St. Petersburg, FL (D.C.); the Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal (H.D.); Tuba City Regional Health Care, Tuba City (C.G., D.H.), and Phoenix Children\u2019s Hospital, Phoenix (H.K.M.) \u2014 both in Arizona; the Department of Pediatrics, Universi..."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"150","reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2013.09.044"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2018.08.027"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2014.9132"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2018-2300"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.4049\/jimmunol.168.12.6323"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.iac.2009.10.004"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-03-840702"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.dnarep.2005.02.001"},{"key":"e_1_3_5_10_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1702831"},{"key":"e_1_3_5_11_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2013-01-476432"},{"key":"e_1_3_5_12_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2027675"},{"key":"e_1_3_5_13_2","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2020.608653"},{"key":"e_1_3_5_14_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1815408"},{"key":"e_1_3_5_15_2","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aad8856"},{"key":"e_1_3_5_16_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2016.064"},{"key":"e_1_3_5_17_2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0608130103"},{"key":"e_1_3_5_18_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2009.162"},{"key":"e_1_3_5_19_2","doi-asserted-by":"publisher","DOI":"10.1089\/dna.2011.1244"},{"key":"e_1_3_5_20_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2013.08.014"},{"key":"e_1_3_5_21_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.emboj.7600206"},{"key":"e_1_3_5_22_2","doi-asserted-by":"publisher","DOI":"10.1002\/humu.21168"},{"key":"e_1_3_5_23_2","doi-asserted-by":"publisher","DOI":"10.1001\/archderm.135.8.927"},{"key":"e_1_3_5_24_2","doi-asserted-by":"publisher","DOI":"10.1155\/2015\/352934"},{"key":"e_1_3_5_25_2","doi-asserted-by":"publisher","DOI":"10.1126\/sciimmunol.abi7160"},{"key":"e_1_3_5_26_2","doi-asserted-by":"publisher","DOI":"10.1137\/1104033"},{"key":"e_1_3_5_27_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2011.20"},{"key":"e_1_3_5_28_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2009.16"},{"key":"e_1_3_5_29_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2010-09-306761"},{"key":"e_1_3_5_30_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2014.062"},{"key":"e_1_3_5_31_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2007.03.009"},{"key":"e_1_3_5_32_2","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2019000298"},{"key":"e_1_3_5_33_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1702011"},{"key":"e_1_3_5_34_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2017.12.782"}],"container-title":["New England Journal of Medicine"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.nejm.org\/doi\/pdf\/10.1056\/NEJMoa2206575","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,6,11]],"date-time":"2025-06-11T23:02:57Z","timestamp":1749682977000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.nejm.org\/doi\/10.1056\/NEJMoa2206575"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,12,22]]},"references-count":33,"journal-issue":{"issue":"25","published-print":{"date-parts":[[2022,12,22]]}},"alternative-id":["10.1056\/NEJMoa2206575"],"URL":"https:\/\/doi.org\/10.1056\/nejmoa2206575","relation":{},"ISSN":["0028-4793","1533-4406"],"issn-type":[{"value":"0028-4793","type":"print"},{"value":"1533-4406","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,12,22]]}}}