AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains-Reference-Cited by-同舟云学术

AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains

Published:2020-06 Issue: Volume:17 Page:174-187
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Hocquemiller Michaël,Hemsley Kim M.,Douglass Meghan L.,Tamang Sarah J.,Neumann Daniel,King Barbara M.,Beard Helen,Trim Paul J.,Winner Leanne K.,Lau Adeline A.,Snel Marten F.,Gomila Cathy,Ausseil Jérôme,Mei Xin,Giersch Laura,Plavsic Mark,Laufer Ralph

Funder

Lysogene

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference58 articles.

1. Mucopolysaccharidosis type IIIA: clinical spectrum and genotype-phenotype correlations;Valstar;Ann. Neurol.,2010

2. Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and Greece;Héron;Am. J. Med. Genet. A.,2011

3. Mucopolysaccharidosis type III (Sanfilippo syndrome): emerging treatment strategies;de Ruijter;Curr. Pharm. Biotechnol.,2011

4. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes;Foust;Nat. Biotechnol.,2009

5. Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain;Deverman;Nat. Biotechnol.,2016

Cited by 23 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Evaluation of neuroretina following i.v. or intra‐CSF AAV9 gene replacement in mice with MPS IIIA, a childhood dementia;CNS Neuroscience & Therapeutics;2024-08

2. Neurosurgical gene therapy for central nervous system diseases;Neurotherapeutics;2024-07

3. Photobiomodulation in the infrared spectrum reverses the expansion of circulating natural killer cells and brain microglial activation in Sanfilippo mice;Journal of Neurochemistry;2024-06-07

4. Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid;Human Gene Therapy;2023-10-23

5. The ice age – A review on formulation of Adeno-associated virus therapeutics;European Journal of Pharmaceutics and Biopharmaceutics;2023-09