中国神经再生研究(英文版) ›› 2023, Vol. 18 ›› Issue (9): 1954-1955.doi: 10.4103/1673-5374.367845

• 观点:退行性病与再生 • 上一篇    下一篇

粘多糖疾病的先天免疫

  

  • 出版日期:2023-09-15 发布日期:2023-03-06

Gene-modified neural progenitor cells for the treatment of neuropathic lysosomal storage diseases

Oriana Mandolfo, Brian W. Bigger*   

  1. Stem Cell and Neurotherapies, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK
  • Online:2023-09-15 Published:2023-03-06
  • Contact: Brian W. Bigger, PhD, brian.bigger@manchester.ac.uk.
  • Supported by:
    Prof Brian W. Bigger acknowledges support from Innovate UK (Innovate Manchester Advanced Therapy Centre Hub – iMATCH).

摘要: https://orcid.org/0000-0002-9708-1112 (Brian W. Bigger) 

Abstract: Lysosomal storage diseases: Lysosomal storage diseases (LSDs) are a family of about 70 disorders, with an overall incidence of 1:7000 live births. They are caused by dysfunctional lysosomal hydrolases, eventually leading to the accumulation of undegraded substrate into the lysosome. This results in a wide array of symptoms, which may include: the presence of dysmorphic features, cardio-respiratory disease, bone and joint disease, organomegaly, developmental delay and neurocognitive decline. The majority of these diseases have a neurological component and in the absence of treatment, death often occurs in the first decades of life, with the neurological complications drastically undermining the patient’s quality of life, as well as their families (Boustany, 2013).