Widespread correction of brain pathology in feline alpha-mannosidosis by dose escalation of intracisternal AAV vector injection

Author:

Hunter Jacqueline E.,Molony Caitlyn M.,Bagel Jessica H.,O’Donnell Patricia,Vite Charles H.,Chawla Sanjeev,Poptani Harish,Wolfe John H.ORCID

Funder

NIH

Publisher

Elsevier BV

Reference43 articles.

1. Alpha-mannosidosis;Malm;Orphanet J. Rare Dis.,2008

2. The natural course and complications of alpha-mannosidosis—a retrospective and descriptive study;Malm;J. Inherit. Metab. Dis.,2014

3. Disorders of glycoprotein degradation: α-mannosidosis, β- mannosidosis, fucosidosis, sialidosis, and aspartylglycosaminuria;Wolfe,2024

4. Alpha-Mannosidosis: Therapeutic Strategies;Ceccarini;Int. J. Mol. Sci.,2018

5. Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial;Borgwardt;J. Inherit. Metab. Dis.,2018

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