Výsledky vyhledávání - "therapeutic use [Antibodies, Neutralizing]"

Prevalence of Anti-Adeno-Associated Virus Serotype 9 Antibodies in Adult Patients with Spinal Muscular Atrophy

Autor: Benjamin Stolte, Olivia Schreiber-Katz, René Günther, Claudia Diana Wurster, Susanne Petri, Alma Osmanovic, Maren Freigang, Zeljko Uzelac, Markus Leo, Otgonzul von Velsen, Wibke Bayer, Ulf Dittmer, Christoph Kleinschnitz, Tim Hagenacker

Publikováno v: Human gene therapy 33, 968-976 (2022). doi:10.1089/hum.2022.054

5q-associated spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder that leads to progressive muscle atrophy and weakness. The disease is caused by a homozygous deletion or mutation in the survival of motor neuron 1 (SMN1) ge

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::68a249cf634b6e20bf7773fab7e41bb5
https://pub.dzne.de/record/165149

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