An update on gene therapy for lysosomal storage disorders

@article{Nagree2019AnUO,
  title={An update on gene therapy for lysosomal storage disorders},
  author={Murtaza S Nagree and Simone Scalia and William M McKillop and Jeffrey A. Medin},
  journal={Expert Opinion on Biological Therapy},
  year={2019},
  volume={19},
  pages={655 - 670}
}
ABSTRACT Introduction: Gene therapies can be envisioned for many disorders where conventional therapies fall short. Lysosomal Storage Disorders (LSDs) are inherited, mostly monogenic, disorders resulting from deficient lysosomal enzyme or co-factor activity. Existing standard-of-care treatments for LSDs are expensive and can negatively impact quality-of-life. They also may not be sufficiently efficacious. LSDs are particularly amenable to gene therapy as modified cells can secrete functional… 

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