Tag: gene therapy

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

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

August 24, 2023/Hematology & oncology

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


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Novel Therapeutics Clinic
February 10, 2023/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

Aleksandra Rachitskaya, MD, delivers investigational gene therapy during pars plana vitrectomy.
June 22, 2022/Eye Care & Treatment

More Gene Therapy Options Available for Inherited Retinal Diseases

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

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

650×450-Sickle Cell Trait
March 23, 2020/News & Insights

Advances in Sickle Cell Disease Bring Additional Therapeutic and Curative Options

Disease-modifying medications, haploidentical transplant and gene therapies

December 8, 2016/Ophthalmology Research

Gene Therapy for Inherited Retinal Diseases

Treatments for retinal dystrophies are in sight

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