Gene therapy shows promise for treating Niemann-Pick disease type C1
Por um escritor misterioso
Descrição
NIH researchers demonstrated that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1.
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://columbuschildren.org/wp-content/uploads/2022/04/AADC-NewsObserver.jpeg)
delta – Columbus Children's Foundation
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://media.springernature.com/m685/springer-static/image/art%3A10.1186%2F1750-1172-5-16/MediaObjects/13023_2009_Article_179_Fig1_HTML.jpg)
Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://pub.mdpi-res.com/ijms/ijms-22-06600/article_deploy/html/images/ijms-22-06600-g001.png?1624179020)
IJMS, Free Full-Text
![Gene therapy shows promise for treating Niemann-Pick disease type C1](http://thescienceexplorer.com/sites/thescienceexplorer.com/files/styles/content_image__large/public/blog/neimann-pick-disease-oc21.jpg?itok=DK4ySYXu×tamp=1477083571)
A Potential New Treatment for Niemann-Pick Disease – ASHG 2016
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://scx2.b-cdn.net/gfx/news/hires/2021/researchers-find-immun.jpg)
Researchers find immune component to rare neurodegenerative disease
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://pubs.acs.org/cms/10.1021/acs.molpharmaceut.2c00463/asset/images/large/mp2c00463_0004.jpeg)
mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://ars.els-cdn.com/content/image/1-s2.0-S1388198119300496-gr1.jpg)
The complexity of a monogenic neurodegenerative disease: More than two decades of therapeutic driven research into Niemann-Pick type C disease - ScienceDirect
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://www.medindia.net/images/common/health-tips/450_237/new-hope-for-patients-with-niemann-pick-c-disorder.jpg)
Health Tip on New Treatment that Shows Promise in Niemann-Pick Type C Disorder - Health Tips
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://www.frontiersin.org/files/Articles/695937/fnmol-14-695937-HTML-r1/image_m/fnmol-14-695937-t002.jpg)
Frontiers Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord
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