Counteracting the Common Shwachman–Diamond Syndrome-Causing SBDS c.258+2T>C Mutation by RNA Therapeutics and Base/Prime Editing

Shwachman–Diamond syndrome (SDS) represents one of the most common inherited bone marrow failure syndromes and is mainly caused by SBDS gene mutations. Only supportive treatments are available, with hematopoietic cell transplantation required when marrow failure occurs. Among all causative mutations...

Descripción completa

Detalles Bibliográficos
Autores principales: Peretto, Laura, Tonetto, Elena, Maestri, Iva, Bezzerri, Valentino, Valli, Roberto, Cipolli, Marco, Pinotti, Mirko, Balestra, Dario
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9962285/
https://www.ncbi.nlm.nih.gov/pubmed/36835434
http://dx.doi.org/10.3390/ijms24044024

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9962285/
https://www.ncbi.nlm.nih.gov/pubmed/36835434
http://dx.doi.org/10.3390/ijms24044024

Ejemplares similares