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Spinal muscular atrophy

First Spliceosome Blueprint Reveals Complex Interplay, Uncovers Drug Targets
Cancer

First Spliceosome Blueprint Reveals Complex Interplay, Uncovers Drug Targets

Alternative Splicing’s Unproductive Transcripts Eliminated by Nonsense-Mediated Decay
Featured

Alternative Splicing’s Unproductive Transcripts Eliminated by Nonsense-Mediated Decay

Spinal Muscular Atrophy Organoid Study Points to Early Neurodevelopmental Defects
Featured

Spinal Muscular Atrophy Organoid Study Points to Early Neurodevelopmental Defects

ASGCT 2024: David Liu Touts Rapid Evolution of Gene-Sized Programmable Editing Technologies
Genome Editing

ASGCT 2024: David Liu Touts Rapid Evolution of Gene-Sized Programmable Editing Technologies

Pancreatic Cancer Drugs Could Reflect Alternative Splicing Signatures
Cancer

Pancreatic Cancer Drugs Could Reflect Alternative Splicing Signatures

Novartis, Voyager Ink Up-to-$1.3B Expansion of Neuro Gene Therapy Collaboration
Drug Discovery

Novartis, Voyager Ink Up-to-$1.3B Expansion of Neuro Gene Therapy Collaboration

Neuromuscular Junction Model Developed Using Pluripotent Stem Cells
News

Neuromuscular Junction Model Developed Using Pluripotent Stem Cells

Base Editing Strategy for Spinal Muscular Atrophy Rescues Patient Fibroblasts, Reverses Mouse Pathology
Drug Discovery

Base Editing Strategy for Spinal Muscular Atrophy Rescues Patient Fibroblasts, Reverses Mouse Pathology

Going Dutch: VectorY’s $138M Series A to Advance Vectorized Antibody Programs for Neurodegenerative Diseases
Drug Discovery

Going Dutch: VectorY’s $138M Series A to Advance Vectorized Antibody Programs for Neurodegenerative Diseases

Antibodies Take Center Stage in Alzheimer’s
Drug Discovery

Antibodies Take Center Stage in Alzheimer’s

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