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Archives: Publication

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction.

February 19, 2021

AAV-8 and AAV-9 Vectors Cooperate with Serum Proteins Differently Than AAV-1 and AAV-6.

February 19, 2021

Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.

February 19, 2021

Repairing folding-defective alpha-sarcoglycan mutants by CFTR correctors, a potential therapy for Limb Girdle Muscular Dystrophy 2D.

February 19, 2021

Emerging Issues in AAV-Mediated In Vivo Gene Therapy.

February 19, 2021

Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice.

February 19, 2021

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human beta-globin locus.

February 19, 2021

Loss of Sarcomeric Scaffolding as a Common Baseline Histopathologic Lesion in Titin-Related Myopathies.

February 19, 2021

Diagnostic anoctamin-5 protein defect in patients with ANO5-mutated muscular dystrophy.

February 19, 2021

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

February 19, 2021

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