CRISPR Milestone and Gene Therapy Redefine the Curative Landscape of Beta-Thalassemia
A new review charts how stem cell transplantation, lentiviral gene therapy, and CRISPR-based editing such as Casgevy are turning beta-thalassemia ...
Tag: gene therapy
Optogenetic Therapy Shows Signs of Restoring Vision in Advanced Blindness
An international trial led by University of Pittsburgh scientists found that an optogenetic gene therapy combined with light-projecting goggles was ...
Telomerase Enzyme Emerges as a Drug Target for Healing the Brain After Stroke
New research shows that the telomerase enzyme TERT drives neural repair after stroke and that the herbal compound cycloastragenol can ...
Patient Tumors Grown From Stem Cells Become Living Test Beds for Duchenne Gene Editing
Researchers converted Duchenne patient stem-cell teratomas into humanized models in which both gene-corrected cell transplants and lipid nanoparticle base editing ...
Topical Gene Therapy B-VEC Offers New Hope for Butterfly Skin Disease
A new review confirms that beremagene geperpavec, the first FDA-approved topical gene therapy, significantly improves wound healing in dystrophic epidermolysis ...
Silencing HPV16 Oncogenes E5 and E6 Emerges as a Promising Route Against Cervical Cancer
A new review in Virology Journal examines how siRNA and CRISPR/Cas gene-silencing technologies could target the HPV16 oncoproteins E5 and ...
Activated Protein C Keeps Blood Stem Cells Resting and Boosts Transplant Success
University of Zurich researchers found that a one-hour treatment with activated protein C keeps human blood stem cells in a ...
Million-Dollar Cures: Why Insurance Policy Now Decides Which Children Get Gene Therapy
A new Pediatric Research commentary argues that insurance coverage and reimbursement policy, not the underlying science, now determine which children ...
Gene Therapy With Goggles Lets Blind Patients See Objects, Five-Year Study Shows
A five-year clinical cohort study shows that an optogenetic therapy combining gene injection and light-stimulating goggles is safe and enabled ...
Hidden Protein Networks Emerge as Central Players in Huntington’s Disease
A new review argues that Huntington's disease is driven by widespread disruptions of interconnected protein networks, pointing to hub proteins ...
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Mothers who receive childcare support from maternal grandparents show more parental warmth, finds NTU Singapore study
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University of Seville Breaks 120-Year-Old Mystery, Revises a Key Einstein Concept
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Bee body mass, pathogens and local climate influence heat tolerance
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Researchers record first-ever images and data of a shark experiencing a boat strike
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Groundbreaking Clinical Trial Reveals Lubiprostone Enhances Kidney Function
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