Results 171 to 180 of about 90,357 (220)

New Indications for Hematopoietic Stem Cell Gene Therapy in Lysosomal Storage Disorders. [PDF]

Front Oncol, 2022
Rossini L, Durante C, Marzollo A, Biffi A.   +3 more
europepmc   +1 more source

Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses

, 2008
Chris Pierret, Jason A. Morrison, Mark D. Kirk   +2 more
openalex   +2 more sources

The Unmet Needs of Lysosomal Storage Disorders from Early Diagnosis to Caregiving Pathways: An Italian Perspective. [PDF]

J Clin Med
Castaman G, Linari S, Barbato A, Costantino N, Dionisi-Vici C, Menni F, Procopio E, Ramat S, Torquati F, Verrecchia E, Scarpa M.   +10 more
europepmc   +1 more source

Glycosphingolipids and lysosomal storage disorders as illustrated by gaucher disease.

Current Opinion in Chemical Biology, 2019
J. Aerts, C. Kuo, Lindsey T. Lelieveld, D. Boer, Martijn J. C. van der Lienden, H. Overkleeft, M. Artola   +6 more
semanticscholar   +1 more source

Lysosomal storage disorders as an etiology of nonimmune hydrops fetalis: A systematic review. [PDF]

Clin Genet, 2021
Iyer NS, Gimovsky AC, Ferreira CR, Critchlow E, Al-Kouatly HB.   +4 more
europepmc   +1 more source

Pathogenic mycobacteria achieve cellular persistence by inhibiting the Niemann-Pick Type C disease cellular pathway [version 1; referees: 2 approved, 2 approved with reservations] [PDF]

, 2016
et al,, Ory, Daniel S
core   +1 more source

Correction to Xing M, Parker EI, Moreno-De-Luca A, Harmouche E, Terk MR. Radiological and clinical characterization of the lysosomal storage disorders: non-lipid disorders.Br J Radiol2014;87: 20120467

, 2014
openalex   +2 more sources

Biomarkers for gene therapy clinical trials of lysosomal storage disorders. [PDF]

Mol Ther
Rossi A, Malvagia S, la Marca G, Parenti G, Brunetti-Pierri N.   +4 more
europepmc   +1 more source

Drug-like screening, molecular docking, molecular dynamics simulations, and binding free energies on the interaction of pyrazole derivatives as inhibitors of lysosomal storage disorders and anticancer activity

Emmanuel Israel Edache, Adebiyi Adedayo, Hadiza Adamu Dawi, Fabian Audu Ugbe   +3 more
openalex   +1 more source

Relative acidic compartment volume as a lysosomal storage disorder–associated biomarker

, 2014
Daniëlle te Vruchte, Anneliese O. Speak, Kerri L. Wallom, Nada Al Eisa, David A. Smith, Christian J. Hendriksz, Louise Simmons, Robin Lachmann, Alison Cousins, Ralf Hartung, Eugen Mengel, Heiko Runz, Michael Beck, Yasmina Amraoui, Jackie Imrie, Elizabeth Jacklin, Kate Riddick, Nicole M. Yanjanin, Christopher A. Wassif, Arndt Rolfs, Florian Rimmele, Naomi Wright, Clare Taylor, Uma Ramaswami, Timothy M. Cox, Caroline A. Hastings, Xuntian Jiang, Rohini Sidhu, Daniel S. Ory, Begoña Arias, Mylvaganam Jeyakumar, Daniel J. Sillence, J. E. Wraith, Forbes D. Porter, Mario Cortina‐Borja, Frances M. Platt   +35 more
openalex   +2 more sources