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Tag: gene therapy

gene editing

Novel Gene Editing Therapy for Sickle Cell Disease Continues to Free Patients From Severe Vaso-Occlusive Pain

Interim results of RUBY study also indicate improved physical function and quality of life

Dr. Imad Najm against a decorative background
September 16, 2024/Neurosciences/Podcast

Temporal Lobe Epilepsy and the Potential of Gene Therapy (Podcast)

Investigational gene approaches offer hope for a therapeutically challenging condition

CQD-CHP4445460-hanna-sickle-650×450
January 30, 2024/Cancer

Gene Therapy Trials Show Positive Results in Sickle Cell Disease and Thalassemia

First-in-human trials of CRISPR-Cas12a gene editing demonstrate safety and meaningful event-free survival

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HVI_Kelley_4277155_Patient Procedure Shoot- First in Human Trial
October 27, 2023/Genomic Medicine

Cleveland Clinic Treats First Patient With Investigational Gene Therapy for Hypertrophic Cardiomyopathy

One-time infusion of adenovirus-based therapy is designed to restore heart muscle function

SCD

Trial of Gene Therapy to Treat Sickle Cell Disease Reports Initial Positive Results

First in-human trial using CRISPR/CASP 12 for gene editing in sickle cell disease

Novel Therapeutics Clinic
February 10, 2023/Cancer/Research

Novel Therapeutics Clinic Offers Access to Early-Phase Trials for Solid Tumors

Dedicated investigators and clinicians bring expertise in drug development and flexible study design

Eye with DNA helix

More Gene Therapy Options Available for Inherited Retinal Diseases

Trial enrolling now for patients with retinitis pigmentosa caused by RPGR mutation

Genetics brain concept
December 17, 2021/Genomic Medicine

The Promise of Gene Therapy in Neurological Disorders (Podcast)

Advanced genomic research techniques show potential to treat a variety of conditions

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