Treatment of a Mouse Model of ALS by In Vivo Base Editing

Amyotrophic lateral sclerosis (ALS) is a debilitating and fatal disorder that can be caused by mutations in the superoxide dismutase 1 (SOD1) gene. Although ALS is currently incurable, CRISPR base editors hold the potential to treat the disease through their ability to create nonsense mutations that...

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Veröffentlicht in:	Molecular therapy 2020-04, Vol.28 (4), p.1177-1189
Hauptverfasser:	Lim, Colin K.W., Gapinske, Michael, Brooks, Alexandra K., Woods, Wendy S., Powell, Jackson E., Zeballos C., M. Alejandra, Winter, Jackson, Perez-Pinera, Pablo, Gaj, Thomas
Format:	Artikel
Sprache:	eng
Schlagworte:	AAV ALS Amyotrophic Lateral Sclerosis - genetics Amyotrophic Lateral Sclerosis - therapy Animals base editor Biotechnology & Applied Microbiology Codon, Nonsense CRISPR-Associated Protein 9 - metabolism CRISPR-Cas9 Dependovirus - genetics Disease Models, Animal Gene Editing gene therapy Genetic Vectors - administration & dosage Genetics & Heredity HEK293 Cells Humans Injections, Spinal Inteins Life Sciences & Biomedicine Male Medicine, Research & Experimental Mice Mice, Transgenic neurodegeneration Original Research & Experimental Medicine Science & Technology SOD1 split intein Streptococcus pyogenes - enzymology Superoxide Dismutase-1 - genetics Trans-Splicing Treatment Outcome
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