Show simple item record

dc.contributor.authorGruntman, Alisha
dc.contributor.authorFlotte, Terence R.
dc.date2022-08-11T08:10:13.000
dc.date.accessioned2022-08-23T16:59:55Z
dc.date.available2022-08-23T16:59:55Z
dc.date.issued2018-04-01
dc.date.submitted2019-08-30
dc.identifier.citation<p>FASEB J. 2018 Apr;32(4):1733-1740. doi: 10.1096/fj.201700982R. <a href="https://doi.org/10.1096/fj.201700982R">Link to article on publisher's site</a></p>
dc.identifier.issn0892-6638 (Linking)
dc.identifier.doi10.1096/fj.201700982R
dc.identifier.pmid31282760
dc.identifier.urihttp://hdl.handle.net/20.500.14038/43696
dc.description.abstractGene therapy is emerging as a viable option for clinical therapy of monogenic disorders and other genetically defined diseases, with approved gene therapies available in Europe and newly approved gene therapies in the United States. In the past 10 years, gene therapy has moved from a distant possibility, even in the minds of much of the scientific community, to being widely realized as a valuable therapeutic tool with wide-ranging potential. The U.S. Food and Drug Administration has recently approved Luxturna (Spark Therapeutics Inc, Philadelphia, PA, USA), a recombinant adeno-associated virus (rAAV) 2 gene therapy for one type of Leber congenital amaurosis 2 ( 1 , 2 ). The European Medicines Agency (EMA) has approved 3 recombinant viral vector products: Glybera (UniQure, Amsterdam, The Netherlands), an rAAV vector for lipoprotein lipase deficiency; Strimvelis (Glaxo Smith-Kline, Brentford, United Kingdom), an ex vivo gammaretrovirus-based therapy for patients with adenosine deaminase-deficient severe combined immune deficiency (ADA-SCID); and Kymriah (Novartis, Basel, Switzerland), an ex vivo lentivirus-based therapy to engineer autologous chimeric antigen-receptor T (CAR-T) cells targeting CD19-positive cells in acute lymphoblastic leukemia. These examples will be followed by the clinical approval of other gene therapy products as this field matures. In this review we provide an overview of the state of gene therapy by discussing where the field stands with respect to the different gene therapy vector platforms and the types of therapies that are available.
dc.language.isoen_US
dc.relation<p><a href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&list_uids=31282760&dopt=Abstract">Link to Article in PubMed</a></p>
dc.relation.urlhttps://doi.org/10.1096/fj.201700982R
dc.subjectAAV
dc.subjectapproved clinical products
dc.subjectgammaretrovirus
dc.subjectgene editing
dc.subjectlentivirus
dc.subjectvector platforms
dc.subjectCongenital, Hereditary, and Neonatal Diseases and Abnormalities
dc.subjectGenetic Phenomena
dc.subjectGenetics and Genomics
dc.subjectTherapeutics
dc.titleThe rapidly evolving state of gene therapy
dc.typeArticle
dc.source.journaltitleFASEB journal : official publication of the Federation of American Societies for Experimental Biology
dc.source.volume32
dc.source.issue4
dc.identifier.legacycoverpagehttps://escholarship.umassmed.edu/peds_pp/278
dc.identifier.contextkey15236984
html.description.abstract<p>Gene therapy is emerging as a viable option for clinical therapy of monogenic disorders and other genetically defined diseases, with approved gene therapies available in Europe and newly approved gene therapies in the United States. In the past 10 years, gene therapy has moved from a distant possibility, even in the minds of much of the scientific community, to being widely realized as a valuable therapeutic tool with wide-ranging potential. The U.S. Food and Drug Administration has recently approved Luxturna (Spark Therapeutics Inc, Philadelphia, PA, USA), a recombinant adeno-associated virus (rAAV) 2 gene therapy for one type of Leber congenital amaurosis 2 ( 1 , 2 ). The European Medicines Agency (EMA) has approved 3 recombinant viral vector products: Glybera (UniQure, Amsterdam, The Netherlands), an rAAV vector for lipoprotein lipase deficiency; Strimvelis (Glaxo Smith-Kline, Brentford, United Kingdom), an ex vivo gammaretrovirus-based therapy for patients with adenosine deaminase-deficient severe combined immune deficiency (ADA-SCID); and Kymriah (Novartis, Basel, Switzerland), an ex vivo lentivirus-based therapy to engineer autologous chimeric antigen-receptor T (CAR-T) cells targeting CD19-positive cells in acute lymphoblastic leukemia. These examples will be followed by the clinical approval of other gene therapy products as this field matures. In this review we provide an overview of the state of gene therapy by discussing where the field stands with respect to the different gene therapy vector platforms and the types of therapies that are available.</p>
dc.identifier.submissionpathpeds_pp/278
dc.contributor.departmentDepartment of Pediatrics
dc.contributor.departmentHorae Gene Therapy Center
dc.source.pages1733-1740


This item appears in the following Collection(s)

Show simple item record