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Literature DB >> 33743228

Gene therapy for spinal muscular atrophy: hope and caution.

Nathalie Goemans¹.

Abstract

Entities: Disease

Mesh：

Substances：

Year: 2021 PMID： 33743228 DOI： 10.1016/S1474-4422(21)00071-5

Source DB: PubMed Journal: Lancet Neurol ISSN： 1474-4422 Impact factor: 44.182

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3 in total

Review 1. Advances in gene therapy for neurogenetic diseases: a brief review.

Authors: Ying-Xuan Xie; Wen-Qi Lv; Yi-Kun Chen; Shunyan Hong; Xiang-Ping Yao; Wan-Jin Chen; Miao Zhao
Journal: J Mol Med (Berl) Date: 2021-11-27 Impact factor: 4.599

Review 2. Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy.

Authors: Hannah A Blair
Journal: CNS Drugs Date: 2022-08-12 Impact factor: 6.497

Review 3. Precision Medicine Based on CFTR Genotype for People with Cystic Fibrosis.

Authors: Iram Haq; Maryam Almulhem; Simone Soars; David Poulton; Malcolm Brodlie
Journal: Pharmgenomics Pers Med Date: 2022-02-05

3 in total