Gene therapy shows promise for treating Niemann-Pick disease type C1
Por um escritor misterioso
Descrição
NIH researchers demonstrated that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1.
Researchers find immune component to rare neurodegenerative disease
Reality of Rare Diseases: A Unique Perspective About Rare Diseases
Clinical applications of gene therapy for rare diseases: A review - Papaioannou - 2023 - International Journal of Experimental Pathology - Wiley Online Library
Marta, with Niemann Pick C, fights for a cure with gene therapy - Fundación Columbus
Models to study basic and applied aspects of lysosomal storage disorders - ScienceDirect
Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
Frontiers CNS-Targeting Therapies for Lysosomal Storage Diseases: Current Advances and Challenges
A Potential New Treatment for Niemann-Pick Disease – ASHG 2016
delta – Columbus Children's Foundation
de
por adulto (o preço varia de acordo com o tamanho do grupo)