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Initial data indicate tolerability and promising cardiac remodeling effects
Interim results of RUBY study also indicate improved physical function and quality of life
A conversation with Marcelo Pasquini, MD
Investigational gene approaches offer hope for a therapeutically challenging condition
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First-in-human trials of CRISPR-Cas12a gene editing demonstrate safety and meaningful event-free survival
One-time infusion of adenovirus-based therapy is designed to restore heart muscle function
First in-human trial using CRISPR/CASP 12 for gene editing in sickle cell disease
Driving advances in cancer care
Dedicated investigators and clinicians bring expertise in drug development and flexible study design
Trial enrolling now for patients with retinitis pigmentosa caused by RPGR mutation
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