Advances in therapies for neurological Lysosomal Storage Disorders.
Sign Up to like & getrecommendations! Published in 2023 at "Journal of inherited metabolic disease"
DOI: 10.1002/jimd.12615
Abstract: Lysosomal Storage Disorders (LSDs) are a diverse group of inherited, monogenic diseases caused by functional defects in specific lysosomal proteins. The lysosome is a cellular organelle that plays a critical role in catabolism of waste… read more here.
Keywords: therapies neurological; storage disorders; neurological lysosomal; lysosomal storage ... See more keywords
Delivering Hematopoietic Stem Cell Gene Therapy Treatments for Neurological Lysosomal Diseases.
Sign Up to like & getrecommendations! Published in 2019 at "ACS chemical neuroscience"
DOI: 10.1021/acschemneuro.8b00408
Abstract: Neurological lysosomal storage diseases are rare, inherited conditions resulting mainly from lysosomal enzyme deficiencies. Current treatments, such as enzyme replacement therapy and hematopoietic stem cell transplantation, fail to effectively treat neurological disease due to insufficient… read more here.
Keywords: gene therapy; stem cell; hematopoietic stem; therapy ... See more keywords