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#genetherapy

2 posts2 participants0 posts today
Public
Outi 🦊🌱🌍🌈

Doctors in London have become the first in the world to cure blindness in children born with a rare genetic condition using a pioneering gene therapy.

The children had leber congenital amaurosis (LCA), a severe form of retinal dystrophy that causes vision loss due to a defect in the AIPL1 gene.

After treatment, four children can now see shapes, find toys, recognise their parents’ faces, and in some cases, even read and write.

#science #medicine #GeneTherapy

theguardian.com/society/2025/f

The Guardian · Doctors in London cure blindness in children with rare conditionBy Andrew Gregory
Public
Jennifer Tsang

I always love learning about new plasmid technologies (maybe I am biased from my Addgene days). Researchers from the University of Rochester developed a dual AAV vector system called StitchR that can deliver plasmids containing gene fragments that can be subsequently cut out and assembled into a functional gene. This helps overcome the small packaging capacity of AAVs.

Find out more from Drug Discovery News : buff.ly/4hwaxPb

Drug Discover NewsDrug Discovery News, Development & Diagnostics Articles | DDN MagazineToward an AAV gene therapy to treat muscular dystrophiesNew dual AAV vector delivery overcomes challenges in delivering large genes.
#AAV#GeneTherapy
Public *
untsúkonpìiū

$mgtx is currently my biggest single shareholding in my portfolio. Its Phase 3 data on XLRP should come out real soon now. I really love its overall company profile. And the drug really is saving a patient's life. Both Sanofi and JnJ are it's big shareholders. This is very rare.

I will not give my detailed due diligence result here. But this gene therapy company is something you want to follow or even invest.

#investment#biotech#genetherapy
Public
Brittany Trang

I cried reading this story about a dad who stood outside a #genetherapy conference with a poster: “WANTED: a cure for Michael. REWARD: Research Grant.”

Now he helps other parents.

But is he a superhero of the medical system...or yet another victim of it: the proverbial child selling lemonade to cure his own disease?

statnews.com/2024/12/09/pirovo

STAT · This father built a gene therapy for his son. Now comes the harder part: saving others' children, tooTerry Pirovolakis built a gene therapy to save his son from an ultra-rare disease. Now he has become the first stop and last hope for other desperate parents.
#health#healthcare#medicine
Public
Science Updates

Novel Gene Therapy is a Game-Changer for Sickle Cell Patient

"The treatment has been truly transformative for Branden," said Matthew Heeney, MD. "Not only are his blood counts and markers of disease activity essentially normalized, but he can now complete daily activities that most take for granted and tackle new experiences previously beyond his reach..."

#genetics #GeneTherapy #medicine #health #science #research

labroots.com/trending/genetics

LabrootsSickle Cell Patient is Successfully Treated with a Novel Gene Therapy | Genetics And GenomicsSickle cell disease refers to a group of genetic diseases that impact a protein that ferries oxygen through the blood. That protein is called hemoglobin ... | Genetics And Genomics
Public
Science Updates

Researchers are getting better at finding ways to deliver therapeutic genes into cells. But when genes are really large, this can pose major challenges. In a new study, scientists found a way around that problem when dealing with a particularly large gene, to send a gene therapy system successfully into a mouse model.

#GeneTherapy #genetics #science #research #biology #health #medicine #hearing #vision

labroots.com/trending/genetics

LabrootsA New Approach in Usher Syndrome Gene Therapy | Genetics And GenomicsDelivering very large genes to cells can pose a serious challenge, so scientists developed a new method. | Genetics And Genomics
Public
Wesley Bond (OLD ACCOUNT)

Very disappointing news on the Duchenne muscular dystrophy front. Elevidys, the only available gene therapy for DMD has shown no significant improvement in motor function in kids receiving the treatment after 1 year. However, certain secondary outcomes showed some benefit.

It remains to be seen if FDA will allow it to stay on the market in the US, given that there are no other available treatments for DMD.

ajmc.com/view/sarepta-joins-pf

AJMC · Sarepta Joins Pfizer in DMD Gene Therapy Trial FailuresBy Hayden E. Klein
#Duchenne#musculardystrophy#raredisease
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