Gene therapy shows promise for treating Niemann-Pick disease type C1
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://media.springernature.com/m685/springer-static/image/art%3A10.1038%2Fs41598-022-06112-y/MediaObjects/41598_2022_6112_Fig4_HTML.png)
Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://www.mdpi.com/ijms/ijms-21-08979/article_deploy/html/images/ijms-21-08979-g001-550.jpg)
IJMS, Free Full-Text
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://nnpdf.org/wp-content/uploads/2023/05/Bloomsbury-Logo-SM.png)
Community News – NNPDF
![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_0002.jpeg)
mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://www.mdpi.com/ijms/ijms-24-13006/article_deploy/html/images/ijms-24-13006-g001.png)
IJMS, Free Full-Text
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://www.mdpi.com/diseases/diseases-05-00006/article_deploy/html/images/diseases-05-00006-g001.png)
Diseases, Free Full-Text
![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](https://www.tandfonline.com/cms/asset/cc1f1f7b-091d-48eb-b881-7e2d68001dbd/ieop_a_2215386_f0002_oc.jpg)
Full article: Current advancements in therapy for Niemann-Pick disease: progress and pitfalls
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://scx1.b-cdn.net/csz/news/800a/2019/5d5d42561920e.jpg)
Promising gene replacement therapy moves forward
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://i1.rgstatic.net/publication/367639081_Identification_of_cerebral_spinal_fluid_protein_biomarkers_in_Niemann-Pick_disease_type_C1/links/63d9b92d64fc860638009564/largepreview.png)
PDF) Identification of cerebral spinal fluid protein biomarkers in Niemann-Pick disease, type C1
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://d2jx2rerrg6sh3.cloudfront.net/image-handler/picture/2014/7/Gene_Therapy-620x480.jpg)
New gene therapy shows promise in reversing chronic pain
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://journals.sagepub.com/cms/10.1177/1087057114537378/asset/images/large/10.1177_1087057114537378-fig1.jpeg)
Niemann–Pick Disease Type C: Induced Pluripotent Stem Cell–Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy - Daozhan Yu, Manju Swaroop, Mengqiao Wang, Ulrich Baxa, Rongze Yang, Yiping Yan, Turhan
![Gene therapy shows promise for treating Niemann-Pick disease type C1](https://scx2.b-cdn.net/gfx/news/2019/2-genetic.jpg)
Miglustat improves swallowing in children and adolescents with Niemann-Pick type C1 disease
de
por adulto (o preço varia de acordo com o tamanho do grupo)