Lv61
1920 积分 2023-10-07 加入
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
51分钟前
已关闭
Spinal Muscular Atrophy Treatment in Patients Identified by Newborn Screening—A Systematic Review
3小时前
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Spinal Muscular Atrophy: An Evolving Scenario through New Perspectives in Diagnosis and Advances in Therapies
5小时前
已完结
Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor
9天前
已关闭
Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor
9天前
已关闭
An RNA editing strategy rescues gene duplication in a mouse model of MECP2 duplication syndrome and nonhuman primates
2个月前
已完结
Autism-like behaviours and germline transmission in transgenic monkeys overexpressing MeCP2
2个月前
已完结
Whole-brain in vivo base editing reverses behavioral changes in Mef2c-mutant mice
2个月前
已完结
Direct Dorsal Root Ganglia (DRG) Injection in mice for Analysis of Adeno-associated Viral (AAV) Gene Transfer to Peripheral Somatosensory Neurons
2个月前
已完结
Early transgene expression of GALC enzyme in a phase I/II safety, tolerability and efficacy study of PBKR03 in infants with early infantile Krabbe disease (EIKD) (GALax-C)
5个月前
已关闭
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