The page lists 16 projects related to the topic "aav".
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1 | GLYCODIS3 | Genetic correction of glycogen debranching enzyme deficiency in Glycogen Storage disease III: a proof of concept study | 2016 |
2 | MINERVA | Micro-RNAs of neutrophils in renal ANCA-associated vasculitis | 2016 |
3 | REGENETHER | Modeling and treating retinal degenerative disease | 2015 |
4 | MYOCURE | Development of an innovative gene therapy platform to cure rare hereditary muscle disorders | 2016 |
5 | EYEGET | Gene therapy of inherited retinal diseases | 2017 |
6 | GeneVision | Developing a cure for retinitis pigmentosa due to Usher syndrome | 2017 |
7 | VDJtargeting | Engineering T cells and B cells for Immunotherapy using V(D)J recombination | 2017 |
8 | UshTher | Clinical trial of gene therapy with dual AAV vectors for retinitis pigmentosa in patients with Usher syndrome type IB | 2018 |
9 | CureCN | Adeno-Associated Virus Vector-Mediated Liver Gene Therapy for Crigler-Najjar Syndrome | 2018 |
10 | DIAMONDCOR | A molecular approach to treat diabetes mellitus onset dependent coronaropathy | 2018 |
11 | MITOPOMPE | Targeting mitochondrial defects and oxidative stress in Pompe Disease: from pathogenesis to therapy | 2019 |
12 | NEVULA | Understanding selective neuronal vulnerability in Alzheimer’s disease | 2018 |
13 | CG01 | A novel gene therapy for epilepsy | 2018 |
14 | CuRE | Cardiac REgeneration from within | 2019 |
15 | iHEAR | Gene therapy of inherited and acquired hearing loss | 2019 |
16 | inSight | Moving a novel gene therapy paradigm to treat blindness to the market | 2019 |