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Human Gene Therapy|January 31, 2008
Overcoming the innate immune response to small interfering RNAAdam Judge, Ian MacLachlanHuman Gene Therapy|June 17, 2015
The Bacterial Origins of the CRISPR Genome-Editing RevolutionErik J Sontheimer, Rodolphe BarrangouHuman Gene Therapy|June 23, 2026
Hyperthermia-Enhanced Adeno-Associated Virus Gene Therapy: Mechanisms, Delivery Technologies, and Translational PotentialNi-Hsuan Lin, Spyros A Mavropoulos, Kiyotake IshikawaHuman Gene Therapy|April 27, 2002
High translation efficiency is mediated by the encephalomyocarditis virus internal ribosomal entry sites if the natural sequence surrounding the eleventh AUG is retainedJian Qiao, Vincent Roy, Marie Hélène Girard, et al.Human Gene Therapy|June 19, 2026
Adeno-Associated Virus Gene Therapy Translation: Lessons from Early Regulatory MeetingsRodica Stan, Richa Madan Lomash, Oleg A Shchelochkov, et al.Human Gene Therapy|August 1, 1993
In situ retrovirus-mediated gene transfer into dog liverJ E Cardoso, S Branchereau, P R Jeyaraj, et al.Human Gene Therapy|July 7, 2026
Evaluation of the Toxicity and Efficacy of an Adeno-Associated Viral Vector Expressing BEST1 Delivered by Subretinal Injection in a Canine Model of Human BestrophinopathyAlexa Gray, Jennifer C Kwok, Yu Sato, et al.Human Gene Therapy|July 27, 2026
High Prevalence of Preexisting Neutralizing Antibodies Against Adeno-Associated Virus Capsids in Mongolian People with HemophiliaNemekhbayar Baatartsogt, Khongorzul Batchuluun, Yuji Kashiwakura, et al.Human Gene Therapy|July 27, 2026
Systematic Benchmarking of CRISPR-Cas9 Off-Target Prediction Tools Reveals Limitations and Implications for Preclinical AssessmentMasako M Kaufmann, Maren Hackenberg, William Jobson Pargeter, et al.Human Gene Therapy|August 3, 2026
Adeno-Associated Virus-Mediated Central Nervous System Gene Transfer to Suppress Alzheimer's Disease High-Risk APOE4 Variant and Replace with Protective APOE2Kalpita R Karan, Neil R Hackett, Ronald G CrystalPageof 413