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Gene therapy with the TRF1 telomere gene rescues decreased TRF1 levels with aging and prolongs mouse health span
journal article
Generation and characterization of a human iPSC cell line expressing inducible Cas9 in the "safe harbor" AAVS1 locus
Telomerase gene therapy rescues telomere length, bone marrow aplasia, and survival in mice with aplastic anemia.
Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients
Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy.