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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

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

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

ASGCT News: Siren Biotechnology Hails Universal Gene Therapy for Oncology
Cancer

ASGCT News: Siren Biotechnology Hails Universal Gene Therapy for Oncology

Antisense Oligonucleotides Help Treat Deadly Brain Cancer in Mice
Cancer

Antisense Oligonucleotides Help Treat Deadly Brain Cancer in Mice

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

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

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

Challenges in Biologics R&D
Bioprocessing

Challenges in Biologics R&D

Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma
Genome Editing

Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma

Scholar Rock: A New Twist on Targeting Growth Factors
Cancer

Scholar Rock: A New Twist on Targeting Growth Factors

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

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