Results 101 to 110 of about 3,656,183 (130)

Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases

Molecular Therapy - Methods and Clinical Development, 2022
Deni S Galileo, Shunji Tomatsu, Grzegorz Węgrzyn   +2 more
exaly  

[Lysosomal storage disease].

Nihon rinsho. Japanese journal of clinical medicine, 1996
Lysosomes are the principal sites of intracellular digestion. In Lysosomes approximately 40 hydrolytic enzymes are contained. Lysosomal storage diseases are mainly caused by genetic defects that affect one or more of the lysosomal hydrolases, and result in accumulation of their undigested substrates in lysosomes, with profound pathological consequences.
openaire   +1 more source

Lysosomal storage diseases

Pharmacology & Therapeutics, 1982
openaire   +2 more sources

Potential Treatment of Lysosomal Storage Disease through Modulation of the Mitochondrial—Lysosomal Axis

Cells, 2021
Joon Tae Park, Myeong Uk Kuk
exaly  

Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics

EMBO Molecular Medicine, 2009
Giancarlo Parenti
exaly  

Lysosomal storage disorders and Parkinson's disease: Gaucher disease and beyond†‡§

Movement Disorders, 2011
Anthony H. Futerman, Annick Raas-Rothschild   +1 more
exaly  

Lysosomal Storage Disorders Shed Light on Lysosomal Dysfunction in Parkinson’s Disease

International Journal of Molecular Sciences, 2020
Anthony H. Futerman, Shani Blumenreich, Or B Barav   +2 more
exaly  

Lysosomal storage diseases.

RN, 2008
Linda P, Manwaring, Patricia A, Jamerson, Rachel, Slaugh   +2 more
openaire   +3 more sources

Lysosomal Ca homeostasis: Role in pathogenesis of lysosomal storage diseases

Cell Calcium, 2011
Frances Platt, Emyr Lloyd-Evans
exaly  

Lysosomal storage disorders: The cellular impact of lysosomal dysfunction

Journal of Cell Biology, 2012
Frances Platt, Aarnoud C van der Spoel, Barry Boland   +2 more
exaly