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Archives: Publication

An AAV-SGCG Dose-Response Study in a gamma-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress.

February 19, 2021

Induction of tumor-specific CTL responses using the C-terminal fragment of Viral protein R as cell penetrating peptide.

February 19, 2021

SINEUP Non-coding RNA Targeting GDNF Rescues Motor Deficits and Neurodegeneration in a Mouse Model of Parkinson’s Disease.

February 19, 2021

Functional and cellular localization diversity associated with Fukutin-related protein patient genetic variants.

February 19, 2021

AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy.

February 19, 2021

Temporary Reduction of Membrane CD4 with the Antioxidant MnTBAP Is Sufficient to Prevent Immune Responses Induced by Gene Transfer.

February 19, 2021

Gene Therapy for Pompe Disease: The Time is now.

February 19, 2021

Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

February 19, 2021

Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID.

February 19, 2021

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice.

February 19, 2021

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