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Studies in several immunodeficiency diseases in the past 15 years have demonstrated clear proof of principle that gene therapy can have long-lasting, potentially curative effects without the need to search for allogeneic donors and without risk of graft-versus-host disease. Improvement in gene transfer efficiency for target HSC\/Ps brought to light issues of insertional mutagenesis caused by transfer vectors, resulting in oncogene transactivation and leukemias. Lessons from these adverse events have now led to a new generation of vectors, refinements in conditioning regimens, and manufacturing, which are paving the way for expanded applications of the current technology and recent emphasis on gene targeting\/genome editing as the next advancements in the field.<\/jats:p>","DOI":"10.5966\/sctm.2013-0206","type":"journal-article","created":{"date-parts":[[2014,3,29]],"date-time":"2014-03-29T02:11:06Z","timestamp":1396059066000},"page":"636-642","source":"Crossref","is-referenced-by-count":55,"title":["Concise Review: Lessons Learned From Clinical Trials of Gene Therapy in Monogenic Immunodeficiency Diseases"],"prefix":"10.1093","volume":"3","author":[{"given":"David A.","family":"Williams","sequence":"first","affiliation":[{"name":"Division of Hematology\/Oncology, Boston Children's Hospital, and Department of Pediatric Oncology, Dana Farber Cancer Institute, Harvard Medical School, Harvard Stem Cell Institute, Boston, Massachusetts, 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