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Study uses brain's glymphatic system to deliver gene therapy across blood-brain barrier

A new study describes a gene therapy strategy that uses the brain's own glymphatic transport system to distribute engineered viral vectors throughout the brain.

Newssciencehealthneurology

news-medical.net·Tech by Flipboard·7d ago·5 min read

Precision gene editing technologies: From proof-of-concept to clinical therapies

Insightsciencehealth

Shared by @cp-trendsmolecmed.bsky.social ↗

cell.com16d ago

FDA Clears NIH-Supported Gene Therapy MMA-101 for First Human Trial in Rare Metabolic Disorder

Newssciencehealth

Shared by @NIH ↗

ncats.nih.gov17d ago

Single CRISPR infusion reduces hereditary angioedema attacks by 87% in phase 3 trial

Newssciencehealth

Shared by @biologist077.bsky.social ↗

crisprmedicinenews.com26d ago

One-Time Gene Therapy Restores Vision in 6-Year-Old with Rare Congenital Blindness

Saffie Sandford from Stevenage was diagnosed with Leber's Congenital Amaurosis, (LCA) a mutation in the RPE 65 gene.

Newssciencehealthmedical breakthrough

goodnewsnetwork.org·Tech by Flipboard·26d ago·3 min read

Hematopoietic stem cell gene therapy delivers cell-penetrating frataxin to treat Friedreich's ataxia in preclinical models

Engineered hematopoietic stem cells delivering a secreted, tissue-penetrating frataxin restore mitochondrial function and ameliorate disease features in Friedreich’s ataxia models. This gene and cell therapy approach enables sustained systemic protein del

Newssciencehealthneurologycell.com·Science·27d ago·56 min read

AAV-delivered PICK1 peptide inhibitors provide long-term chronic pain relief in mice

Noes-Holt et al. develop and apply AAV-encoded PICK1 inhibitors that alleviate inflammatory and neuropathic pain in male and female mice. They identify dorsal root ganglia as the site of action, demonstrate regulation of pain-relevant kinases, and confirm

Newssciencehealthneurosciencecell.com·Science·27d ago·97 min read

FDA reconsidering gene therapy for Huntington's disease after official who rejected it leaves agency

Newspoliticshealth

Shared by @jenlucpiquant.bsky.social ↗

arstechnica.com29d ago

ACGT Summit 2026 Marks 25 Years of Progress in Cancer Cell and Gene Therapy

Newssciencehealth

Shared by @acgtfoundation.bsky.social ↗

acgtfoundation.org1mo ago

Gene therapy shows promise for functional HIV cure in monkey study

Newssciencehealth

Shared by @ohsubrain.bsky.social ↗

science.org1mo ago

First human treated in gene therapy trial aiming to reverse cellular aging through partial reprogramming

Newssciencehealth

Shared by @katherinestiles.org ↗

scientificamerican.com1mo ago

High-cost gene therapies face financing barriers in US healthcare system

Insighthealthbusiness

Shared by @schaeffer.usc.edu ↗

statnews.com1mo ago

Life Biosciences' ER-100 gene therapy aims to restore vision by rejuvenating retinal neurons through epigenetic reprogramming

Newssciencehealth

Shared by @amazingmariapian.bsky.social ↗

interestingengineering.com1mo ago

First New York patient cured of sickle cell disease using Lyfgenia gene therapy

For 21 years, Sebastien Beauzile lived with a disease that shaped every part of his existence.

Newssciencehealthmedical breakthroughscienceaim.com·Tech by Flipboard·1mo ago·14 min read

Single Infusion of Gene-Editing Therapy VERVE-102 Shows Promise for Inherited High Cholesterol

Newssciencehealth

Shared by @brain.us.com ↗

discovermagazine.com1mo ago

Eli Lilly's experimental gene-editing therapy VERVE-102 shows promise as one-time treatment for high cholesterol

Newssciencehealth

Shared by @spennington.bsky.social ↗

abcnews.com1mo ago

FDA Approves First Dual-AAV Gene Therapy for Genetic Hearing Loss Under Priority Voucher Program

Newssciencehealth

FDA2mo ago

First In-Vivo Gene-Editing Therapy Successfully Saves Newborn's Life in 2025

Newssciencehealth

popularmechanics.com6mo ago

Alliance for Cancer Gene Therapy5

Study uses brain's glymphatic system to deliver gene therapy across blood-brain barrier

Precision gene editing technologies: From proof-of-concept to clinical therapies

FDA Clears NIH-Supported Gene Therapy MMA-101 for First Human Trial in Rare Metabolic Disorder

Single CRISPR infusion reduces hereditary angioedema attacks by 87% in phase 3 trial

One-Time Gene Therapy Restores Vision in 6-Year-Old with Rare Congenital Blindness

Hematopoietic stem cell gene therapy delivers cell-penetrating frataxin to treat Friedreich's ataxia in preclinical models

AAV-delivered PICK1 peptide inhibitors provide long-term chronic pain relief in mice

FDA reconsidering gene therapy for Huntington's disease after official who rejected it leaves agency

ACGT Summit 2026 Marks 25 Years of Progress in Cancer Cell and Gene Therapy

Gene therapy shows promise for functional HIV cure in monkey study

First human treated in gene therapy trial aiming to reverse cellular aging through partial reprogramming

High-cost gene therapies face financing barriers in US healthcare system

Life Biosciences' ER-100 gene therapy aims to restore vision by rejuvenating retinal neurons through epigenetic reprogramming

First New York patient cured of sickle cell disease using Lyfgenia gene therapy

Single Infusion of Gene-Editing Therapy VERVE-102 Shows Promise for Inherited High Cholesterol

Eli Lilly's experimental gene-editing therapy VERVE-102 shows promise as one-time treatment for high cholesterol

FDA Approves First Dual-AAV Gene Therapy for Genetic Hearing Loss Under Priority Voucher Program

First In-Vivo Gene-Editing Therapy Successfully Saves Newborn's Life in 2025