Latest updates for Gene Therapy

Fresh curated links around gene therapy are collected here so marketers can spot useful updates and turn timely ideas into posts faster.

Recent items include:

  • Gene therapy reverses complete congenital night blindness in mice, improving vision
  • Boston Children’s Hospital – Gene therapy for hearing loss
  • Gene Therapy Reverses Fragile X Traits in Preclinical Study

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Fresh articles and ideas

Recent curated links from global sources. Generate one free draft from any story, then use SocialBu to schedule and refine your content calendar.

bioengineer.org /1 month ago

Gene therapy reverses complete congenital night blindness in mice, improving vision

A new preclinical study suggests that gene augmentation therapy may restore sight in a severe form of inherited night blindness. The work, reported in Gene Therapy, targets complet...

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hospitalsmagazine.com /1 month ago

Boston Children’s Hospital – Gene therapy for hearing loss

In May 2025, a young boy received an injection delivering a healthy copy of a gene called OTOF into the cochlea of his inner ear — one of the first patients with hereditary hearing...

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bioengineer.org /1 month ago

Gene Therapy Reverses Fragile X Traits in Preclinical Study

A gene therapy aimed at correcting fragile X syndrome by restoring the missing FMR1 protein has demonstrated multiple disease-relevant improvements in a mouse model, according to a...

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genengnews.com /1 month ago

Gene Therapy Partners Aim to Enhance Protein Expression

Avenue Biosciences´ machine learning-powered platform engineers and screens signal peptide variants to increase protein secretion from cells. Circio’s circVec technology has demons...

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bioengineer.org /1 month ago

Compact genetic switch offers new potential therapies for drug-resistant epilepsy

Targeted gene therapy is increasingly viewed as a promising route for patients with drug-resistant epilepsy, a condition that affects roughly one-third of people living with epilep...

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medicalxpress.com /3 weeks ago

Single gene injection reverses inherited heart disease in mice and patient-grown tissue

Melbourne researchers have made a gene therapy breakthrough that could restore heart function in children with genetic heart disease, sparing them the need for transplants.

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neurosciencenews.com /1 month ago

Gene Therapy Reverses Severe Traits of Fragile X Syndrome

The therapy restored FMRP expression in crucial brain regions, resulting in reduced audiogenic seizure susceptibility, normalized sensory hyperactivity, and complete reversal of el...

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fiercebiotech.com /2 weeks ago

With mRNA back in the spotlight, tRNA shows potential to treat genetic disease

New research coming out of the University of Toronto has shown that transfer RNA may offer a promising therapeutic avenue for treating genetic diseases.

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genengnews.com /1 day ago

Targeted Lentiviral Delivery Without Vector Reengineering

Rather than requiring genetic modification of the viral glycoprotein, G-Link uses a modular protein adaptor to blind native VSV-G tropism and redirect delivery after vector product...

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biopharmadive.com /3 weeks ago

PTC takes a chance on Sangamo’s Fabry disease gene therapy

Wall Street analysts view the deal as a risky, high-reward bet on a treatment that’s nearing a possible regulatory approval and could be superior to existing medications.

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genengnews.com /3 weeks ago

Rewriting Disease: Oligonucleotides Take Aim at the Untreatable

How synthetic DNA and RNA are becoming the discovery engines behind a new generation of precision therapies. The post Rewriting Disease: Oligonucleotides Take Aim at the Untreatabl...

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medcitynews.com /2 weeks ago

Ultragenyx Gene Therapy Becomes First FDA-Approved Treatment for Ultra-Rare Disease

Ultragenyx Pharmaceutical’s Genglycos received accelerated FDA approval for glycogen storage disease type Ia, an inherited enzyme deficiency. It’s also the first approved gene ther...

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hearingreview.com /1 day ago

Sensorion Receives French Regulatory Approval to Initiate HearConnex Gene Therapy Trial for GJB2-Related Hearing Loss

Sensorion has received French regulatory approval to begin a Phase I/II trial of SENS-601, its investigational gene therapy for GJB2-related hearing loss, with first patient dosing...

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parkinsonsnewstoday.com /1 month ago

New MJFF grant to fund lab studies of one-time Parkinson’s gene therapy

New funding from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) will support lab studies of a one-time gene therapy candidate for Parkinson’s disease. Shape Therapeu...

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genengnews.com /1 week ago

Inhaled Suppressor tRNA Restores Gene Function in Cystic Fibrosis Models

A preclinical study showed how chemically enhanced suppressor tRNAs combined with a lung-targeted delivery system can restore production of a critical protein in models of cystic f...

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channelnewsasia.com /1 month ago

Chinese university investigating girl's death in gene-editing trial

The experimental gene therapy was meant to fix a rare genetic condition that caused a delay in cognitive development.

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bioengineer.org /2 weeks ago

Targeted lipid nanoparticles enable in vivo editing of human blood stem cells

A new study reports a strategy for delivering gene-editing technology directly to human haematopoietic stem cells inside the body, using targeted lipid nanoparticles rather than vi...

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news.mit.edu /3 weeks ago

Tackling rare genetic disorders with patient-focused science

Shannon Knight, a brain and cognitive sciences PhD candidate and McGovern Institute researcher, focuses on developing a novel gene therapy.

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hemophilianewstoday.com /4 weeks ago

Gene and Disease-Modifying Therapies for Hemophilia

Inside the evolving hemophilia treatment landscape, including AAV-based gene therapies, non-factor prophylaxis, and approaches that aim to reduce treatment burden. The post Gene an...

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medicalxpress.com /2 weeks ago

First-in-human pilot trial improves stem cell collection and delivery for sickle cell gene therapy

Patients with sickle cell disease are often hesitant to undergo transformative gene therapy—the current lengthy process requires multiple hospital visits to collect enough stem cel...

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genengnews.com /4 days ago

NewBiologix and Synastra Join Forces to Advance rAAV Manufacturing for Duchenne Gene Therapy

The collaboration brings together Synastra’s expertise in genomic engineering, AAV vector design, rare-disease gene therapy, and translational development with NewBiologix’s propri...

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scmp.com /1 month ago

Chinese scientist faces probe after girl, 6, dies ‘following experimental gene therapy’

A leading Chinese scientist is under investigation by his university after a six-year-old girl reportedly died following experimental gene-editing therapy. The girl, named Mei, die...

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medcitynews.com /3 weeks ago

PTC Therapeutics’ $211M Bid Wins Bankruptcy Auction for Sangamo Gene Therapy

Sangamo Therapeutics filed for Chapter 11 bankruptcy in June after its search for strategic alternatives failed to find a path forward for the company. PTC Therapeutics’ auction wi...

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biopharmadive.com /2 weeks ago

Ultragenyx, after setbacks, nabs first gene therapy approval

The clearance of Genglycos for a rare glycogen storage disease gives Ultragenyx a sellable voucher and momentum ahead of an eagerly anticipated study readout.

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Sources covering Gene Therapy

scmp.com

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bioengineer.org

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feeds.feedburner.com

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hemophilianewstoday.com

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medcitynews.com

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medicalxpress.com

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