Gene therapy for lysosomal storage diseases: the lessons and promise of animal models

Author:

Ellinwood N. Matthew,Vite Charles H.,Haskins Mark E.

Publisher

Wiley

Subject

Genetics (clinical),Drug Discovery,Genetics,Molecular Biology,Molecular Medicine

Cited by 101 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

1. Biochemical characteristics of point mutated <i>Capra hircus</i> lysosome α-mannosidase;Journal of Veterinary Medical Science;2023

2. Newborn screening and the recommended uniform screening panel: Optimal submissions and suggested improvements based on an advocacy organization's decade‐long experience;American Journal of Medical Genetics Part C: Seminars in Medical Genetics;2022-06

3. Monocytes and Macrophages and Their Disorders;Schalm's Veterinary Hematology;2022-03-04

4. Mucopolysaccharidoses;Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics;2021

5. Gene therapy;Advances in Animal Genomics;2021

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