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Fanny Collaud

Universite Paris-Saclay
H指数:22
被引数:1,988

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Gene supplementation therapy restores motor deficits in Gnao1[C215Y] mice and proves broad efficacy in C. elegans model

Gnao1[C215Y]小鼠的基因补充疗法恢复了运动缺陷,并在秀丽隐杆线虫模型中证明了广泛的疗效

2026-07-210
OA
A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease

一种肌动性AAV载体与骨骼肌顺式调控元件相结合,可改善庞贝病小鼠模型的糖原清除。

2025-06-010
OAAI
Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver
2024-05-161
OAAI
Gene Therapy in Patients with the Crigler-Najjar Syndrome

Crigler-najjar综合征患者的基因治疗

2023-08-1726
PRE
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates
2021-11-0422
OAAI
Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects
2020-11-0119
OAAI
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

在抗AAV中和抗体存在下,IgG裂解内肽酶可进行体内基因治疗

2020-06-01214
OAAI
Preclinical development of SPK-3006, an investigational liver-directed AAV gene therapy for the treatment of Pompe disease
2020-02-010
PREAI
Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients

Duchenne肌营养不良症患者对腺相关病毒的体液免疫的患病率和长期监测

2019-08-0140
OAAI
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration
2018-10-05210
OAAI
Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression
2018-03-0132
OAAI
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
2017-11-29104
OAAI
Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors
2017-10-16100
OAAI