Lentiviral Gene Therapy for Artemis-Deficient SCID.
Morton J CowanJason YuJanelle FacchinoCarol Fraser-BrowneUkina SanfordMisako KawaharaJasmeen DaraJanel Long-BoyleJess OhWendy ChanShivali ChagLori BroderickDeepak ChellapandianHélène DecaluweCatherine GolskiDiana HuCaroline Y KuoHolly K MillerAleksandra PetrovicRobert CurrierJoan F HiltonDivya PunwaniChristopher C DvorakHarry L MalechR Scott McIvorJennifer M PuckPublished in: The New England journal of medicine (2022)
Infusion of lentiviral gene-corrected autologous CD34+ cells, preceded by pharmacologically targeted low-exposure busulfan, in infants with newly diagnosed ART-SCID resulted in genetically corrected and functional T and B cells. (Funded by the California Institute for Regenerative Medicine and the National Institute of Allergy and Infectious Diseases; ClinicalTrials.gov number, NCT03538899.).
Keyphrases
- infectious diseases
- newly diagnosed
- induced apoptosis
- copy number
- genome wide
- gene therapy
- genome wide identification
- cell cycle arrest
- low dose
- bone marrow
- hiv infected
- quality improvement
- cancer therapy
- drug delivery
- antiretroviral therapy
- dna methylation
- transcription factor
- cell proliferation
- allogeneic hematopoietic stem cell transplantation
- acute lymphoblastic leukemia
- mesenchymal stem cells