Sage Journals: Discover world-class research

Open access

Case report

First published 2024-03-05

Repeated AAV9 Titer Determination in a Presymptomatic SMA Patient with Three SMN2 Gene Copies – A Case Report

Astrid Eisenkölbl, Manuel Pühringer

Journal of Neuromuscular Diseases

Retricted access

Research article

First published 2023-10

Enhanced Cochlear Transduction by AAV9 with High-Concentration Sucrose

Masao Noda, Ryota Koshu, Mari Shimada Dias [...]

Human Gene Therapy

Open access

Research article

First published 2025-02

Directed Evolution of AAV9 for Efficient Gene Expression in Cardiomyocytes In Vitro and In Vivo

Leonard Hüttermann, Lena C. Schröder, Prithviraj M.V. Shetty [...]

Human Gene Therapy

Retricted access

Research article

First published 2016-8-3

Neurodegeneration-Like Pathological and Behavioral Changes in an AAV9-Mediated p25 Overexpression Mouse Model

Xiao Zhou, Jianou Huang, Suyue Pan [...]

Journal of Alzheimer’s Disease

Retricted access

Research article

First published 2013-05

Strong Cortical and Spinal Cord Transduction After AAV7 and AAV9 Delivery into the Cerebrospinal Fluid of Nonhuman Primates

Lluis Samaranch, Ernesto A. Salegio, Waldy San Sebastian [...]

Human Gene Therapy

Retricted access

Research article

First published 2015-04

Motor Neuron Transduction After Intracisternal Delivery of AAV9 in a Cynomolgus Macaque

Peter Bell, Christian Hinderer, Jean-Pierre Louboutin [...]

Human Gene Therapy Methods (Part B)

Retricted access

Research article

First published 2025-04

Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease,Type C1

Avani V. Mylvara, Cristin D. Davidson, Derek Alexander [...]

Human Gene Therapy

Retricted access

Research article

First published 2018

Selective Neuronal Uptake and Distribution of AAVrh8,AAV9,and AAVrh10 in Sheep After Intra-Striatal Administration

Erica Mondo, Richard Moser, Guangping Gao [...]

Journal of Huntington's Disease

Retricted access

Research article

First published 2016-01

Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease

Phillip A. Doerfler, Adrian G. Todd, Nathalie Clément [...]

Human Gene Therapy

Retricted access

Research article

First published 2019-2

Delivery of Glucosylceramidase Beta Gene Using AAV9 Vector Therapy as a Treatment Strategy in Mouse Models of Gaucher Disease

Sichen Du, Huayuan Ou, Renjie Cui [...]

Human Gene Therapy

Search results