{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T21:15:37Z","timestamp":1777670137296,"version":"3.51.4"},"reference-count":37,"publisher":"SAGE Publications","issue":"3","license":[{"start":{"date-parts":[[2013,11,19]],"date-time":"2013-11-19T00:00:00Z","timestamp":1384819200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Human Gene Therapy"],"published-print":{"date-parts":[[2014,3]]},"abstract":"<jats:p>\n                    Dopamine and serotonin are produced by distinct groups of neurons in the brain, and gene therapies other than direct injection have not been attempted to correct congenital deficiencies in such neurotransmitters. In this study, we performed gene therapy to treat knock-in mice with dopamine and serotonin deficiencies caused by a mutation in the aromatic\n                    <jats:sc>l<\/jats:sc>\n                    -amino acid decarboxylase (\n                    <jats:italic toggle=\"yes\">AADC<\/jats:italic>\n                    ) gene (Ddc\n                    <jats:sup>KI<\/jats:sup>\n                    mice). Intracerebral ventricular injection of neonatal mice with an adeno-associated virus (AAV) serotype 9 (AAV9) vector expressing the human\n                    <jats:italic toggle=\"yes\">AADC<\/jats:italic>\n                    gene (AAV9-hAADC) resulted in widespread AADC expression in the brain. Without treatment, 4-week-old Ddc\n                    <jats:sup>KI<\/jats:sup>\n                    mice exhibited whole-brain homogenate dopamine and serotonin levels of 25% and 15% of normal, respectively. After gene therapy, the levels rose to 100% and 40% of normal, respectively. The gene therapy improved the growth rate and survival of Ddc\n                    <jats:sup>KI<\/jats:sup>\n                    mice and normalized their hindlimb clasping and cardiovascular dysfunctions. The behavioral abnormalities of the Ddc\n                    <jats:sup>KI<\/jats:sup>\n                    mice were partially corrected, and the treated Ddc\n                    <jats:sup>KI<\/jats:sup>\n                    mice were slightly more active than normal mice. No immune reactions resulted from the treatment. Therefore, a congenital neurotransmitter deficiency can be treated safely through inducing widespread expression of the deficient gene in neonatal mice.\n                  <\/jats:p>","DOI":"10.1089\/hum.2013.170","type":"journal-article","created":{"date-parts":[[2013,11,19]],"date-time":"2013-11-19T19:13:30Z","timestamp":1384888410000},"page":"189-198","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":16,"title":["Treatment of Congenital Neurotransmitter Deficiencies by Intracerebral Ventricular Injection of an Adeno-Associated Virus Serotype 9 Vector"],"prefix":"10.1177","volume":"25","author":[{"given":"Ni-Chung","family":"Lee","sequence":"first","affiliation":[{"name":"National Taiwan University Hospital and National Taiwan University College of Medicine","place":["Taiwan"]},{"name":"National Taiwan University Hospital and National Taiwan University College of Medicine","place":["Taiwan"]},{"name":"National Taiwan University College of Medicine","place":["Taiwan"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yin-Hsiu","family":"Chien","sequence":"additional","affiliation":[{"name":"National Taiwan University Hospital and National Taiwan University College of Medicine","place":["Taiwan"]},{"name":"National Taiwan University Hospital and National Taiwan University College of Medicine","place":["Taiwan"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Min-Hsiu","family":"Hu","sequence":"additional","affiliation":[{"name":"National Taiwan University Hospital and National Taiwan University College of Medicine","place":["Taiwan"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Wen-Shin","family":"Liu","sequence":"additional","affiliation":[{"name":"National Taiwan University Hospital and National Taiwan University College of Medicine","place":["Taiwan"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pin-Wen","family":"Chen","sequence":"additional","affiliation":[{"name":"National Taiwan University Hospital and National Taiwan University College of Medicine","place":["Taiwan"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Wei-Hua","family":"Wang","sequence":"additional","affiliation":[{"name":"National Taiwan University Hospital and National Taiwan University College of Medicine","place":["Taiwan"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kai-Yuan","family":"Tzen","sequence":"additional","affiliation":[{"name":"National Taiwan University Hospital and National Taiwan University College of Medicine","place":["Taiwan"]},{"name":"National Taiwan University","place":["Taiwan"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Barry J.","family":"Byrne","sequence":"additional","affiliation":[{"name":"University of Florida"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Wuh-Liang","family":"Hwu","sequence":"additional","affiliation":[{"name":"National Taiwan University Hospital and National Taiwan University College of Medicine","place":["Taiwan"]},{"name":"National Taiwan University Hospital and National Taiwan University College of Medicine","place":["Taiwan"]}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2013,11,19]]},"reference":[{"key":"e_1_3_3_2_1","doi-asserted-by":"publisher","DOI":"10.1023\/A:1005307919767"},{"key":"e_1_3_3_3_1","doi-asserted-by":"publisher","DOI":"10.1023\/A:1005698223186"},{"key":"e_1_3_3_4_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3181e620ae"},{"key":"e_1_3_3_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2008.166"},{"key":"e_1_3_3_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.12065"},{"key":"e_1_3_3_7_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2012.167"},{"key":"e_1_3_3_8_1","doi-asserted-by":"publisher","DOI":"10.1007\/s002130050327"},{"key":"e_1_3_3_9_1","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.3558-04.2004"},{"key":"e_1_3_3_10_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-2002-34497"},{"key":"e_1_3_3_11_1","doi-asserted-by":"publisher","DOI":"10.1038\/nbt.1515"},{"key":"e_1_3_3_12_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2012.200"},{"key":"e_1_3_3_13_1","volume-title":"OMIM Database","author":"Hamosh A.","year":"2011","unstructured":"Hamosh A., , and Mckusick V.A. 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