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Spinal muscular atrophy

Making AAVs the Go-To for Making Gene Therapies Go
Drug Discovery

Making AAVs the Go-To for Making Gene Therapies Go

Kathy Vuksanaj -
0
ASGCT News: David Liu Hails Rapid Progress in Precision Genome Editing
Genome Editing

ASGCT News: David Liu Hails Rapid Progress in Precision Genome Editing

Kathy Vuksanaj -
35
Antisense Oligonucleotides Help Treat Deadly Brain Cancer in Mice
Cancer

Antisense Oligonucleotides Help Treat Deadly Brain Cancer in Mice

Christina Jackson -
0
One-Time Treatment: Base Editing Shows Promise vs. SMA
Genome Editing

One-Time Treatment: Base Editing Shows Promise vs. SMA

Alex Philippidis -
1
Stoked About ASOs: Stoke Therapeutics Treats Rare CNS Diseases With RNA-Based Medicines
GEN Edge

Stoked About ASOs: Stoke Therapeutics Treats Rare CNS Diseases With RNA-Based Medicines

Jonathan D Grinstein, PhD -
0
Challenges in Biologics R&D
Bioprocessing

Challenges in Biologics R&D

Anjali A. Sarkar, PhD -
2
Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma
Genome Editing

Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma

Alex Philippidis -
0
Scholar Rock: A New Twist on Targeting Growth Factors
Cancer

Scholar Rock: A New Twist on Targeting Growth Factors

Malorye Allison Branca -
0
FDA Places Partial Clinical Hold on Some Trials of Novartis Gene Therapy
Genome Editing

FDA Places Partial Clinical Hold on Some Trials of Novartis Gene Therapy

Alex Philippidis -
0
Neuromuscular Platforms for Drug Discovery
Drug Discovery

Neuromuscular Platforms for Drug Discovery

Kathy Vuksanaj -
0
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