Article

Exon7 Targeted CRISPR-Prime Editing Approaches for SMN2 Gene Editing in Spinal Muscular Atrophy (SMA) Disease Can Increase In Vitro SMN Expression

2022-03-21

Abstract excerpt

Spinal Muscular Atrophy (SMA) is a fatal neuromuscular disease characterized by motor neuron loss and advanced muscle weakness, which occurs in functional SMN (Survival Motor Neuron) protein deficiency with SMN1 gene-induced deletions and mutations. The incidence of SMA, which is an autosomal recessive disease, is 1/10,000 in the world. The SMN protein acts as a molecular chaperone in the formation of the spliceo...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 02d26186-912d-53f3-a146-c9da6b93d879
DOI: 10.1101/2022.03.21.484406

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Exon7 Targeted CRISPR-Prime Editing Approaches for SMN2 Gene Editing in Spinal Muscular Atrophy (SMA) Disease Can Increase In Vitro SMN ExpressionDOI 10.1101/2022.03.21.484406Read

Methods for Correction of the Single-Nucleotide Substitution c.840C>T in Exon 7 of the SMN2 Gene.Semantic proximity Read

Targeted-Deletion of a Tiny Sequence via Prime Editing to Restore SMN ExpressionSemantic proximity Read

Disruption of splicing-regulatory elements using CRISPR/Cas9 to rescue spinal muscular atrophy in human iPSCs and miceSemantic proximity Read

Improvement of TaC9-ABE mediated correction of human SMN2 geneSemantic proximity Read

Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent LandscapeSemantic proximity Read

Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophySemantic proximity Read

Seamless Genetic Conversion of SMN2 to SMN1 via CRISPR/Cpf1 and Single-Stranded Oligodeoxynucleotides in Spinal Muscular Atrophy Patient-Specific Induced Pluripotent Stem Cells.Semantic proximity Read

Base editing as a genetic treatment for spinal muscular atrophySemantic proximity Read

Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editingSemantic proximity Read

Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editing.Semantic proximity Read

A Negatively Acting Bifunctional RNA Increases Survival Motor Neuron Both In Vitro and In VivoSemantic proximity Read

Prime Editing Permits the Introduction of Specific Mutations in the Gene Responsible for Duchenne Muscular Dystrophy.Semantic proximity Read

Exon7 Targeted CRISPR-Prime Editing Approaches for <i>SMN2</i> Gene Editing in Spinal Muscular Atrophy (SMA) Disease Can Increase In Vitro SMN Expression

Abstract excerpt

Topics

Identifiers and source

Related research