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The Latest Breaking News from Gene Therapy

Casgevy: World-first gene therapy for sickle cell disease approved in UK

Regulator's approval comes three years after scientists won Nobel Prize for inventing the treatment

Evening Standard

World-first gene therapy for inherited blood disorders approved in UK

The treatment is the first to be licensed using the gene-editing tool known as Crispr, for which its inventors were awarded the Nobel prize in 2020.

The Independent UK

Goodbye Statins? New Gene Editing Therapy Slashes High Cholesterol

A new gene therapy targeting an enzyme involved in familial hypercholesterolemia slashes bad cholesterol in a small clinical trial.

Inverse

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Some children born deaf in China finally able to hear after gene therapy, report says

‘It’s really important that we get the first gene therapy treatment right’

The Independent UK

A new cure for sickle cell disease may be coming. Health advisers will review it next week

There soon may be a new cure for sickle cell disease that attacks the disorder at its genetic source

The Independent UK

Form of gene therapy offers hope for severe alcohol addiction, study finds

US research linked to dopamine release finds macaques decreased consumption by up to 90% after treatment

The Guardian - UK

Gene therapy injection into the brain causes alcohol use disorder to stop — in monkeys

A small, proof-of-concept study in monkeys reveals the potential of a one-off gene therapy to treat people with alcohol use disorder who haven't responded to other treatments.

LiveScience

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Bengaluru-based eye hospital to soon start human clinical trials of indigenous gene therapy for ocular diseases

Narayana Nethralaya to start human clinical trials of indigenous gene therapy for ocular diseases; first such effort in India. Cost of therapy to reduce from ₹7 crore to 1/20th-1/50th of international costs. Meeting of 25 clinicians & scientists held to come out with consensus & establish national registry for rare…

The Hindu

Gene-therapy drops restore teen's vision after genetic disease left his eyes clouded with scars

A teen with dystrophic epidermolysis bullosa, or "butterfly disease," was legally blind but has now had his sight restored with gene therapy.

LiveScience

Gene therapy eyedrops restored a boy's sight. Similar treatments could help millions

Antonio Vento Carvajal has been legally blind for much of his 14 years

The Independent UK

Harvard scientists have identified a drug combo that may reverse aging in just one week: ‘A step towards affordable whole-body rejuvenation'

The “chemical cocktail” helped reverse aging in mice, but some experts are skeptical about whether it will work in humans.

Fortune

Gene therapy for severe hemophilia is approved by FDA

U.S. health regulators have approved a gene therapy for the most common form of hemophilia

The Independent UK

Up First: Titan implosion; 1 year after Roe; Evan Gershkovich's appeal rejected

All five people aboard the Titan are believed dead after an implosion. Saturday marks a year since the Supreme Court overturned Roe. The FDA approves the first gene therapy for muscular dystrophy.

WEKU

First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids

The U.S. Food and Drug Administration has approved the first gene therapy to treat a deadly form of muscular dystrophy

The Independent UK

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The Hindu

Laurus Labs, IIT-Kanpur to collaborate for novel gene therapy

The drug manufacturer will also fund the clinical trials and launch the products in India and emerging markets. Additionally, it will establish a GMP (good manufacturing practice) facility at the Techno Park facility of IIT-K, Laurus said on Monday.

LiveScience

Gene therapy could be used as birth control for cats, small study suggests

A new study shows that gene therapy could be an effective form of birth control for cats.

Inverse

This Cutting-Edge Gene Therapy Could Revolutionize Birth Control — But It's Not for Humans

A new gene therapy that modifies the production of a hormone in cats could make spaying wild cats far easier.

Inverse

This Humble Virus Could Revolutionize How We Treat Genetic Diseases

A new type of gene therapy could be cheaper, safer, and more efficient at treating diseases like muscular dystrophy and sickle cell disease.

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LiveScience

New gene therapy gel is the 1st approved treatment for rare and painful 'butterfly disease'

The U.S. Food and Drug Administration approved a gene therapy for dystrophic epidermolysis bullosa, also known as butterfly disease.

Inverse

Gene Therapy is Upending How Doctors Treat Incurable Conditions

Gene therapy has helped doctors make major strides in treating inherited blindness, and it's now being explored for other conditions.

The Conversation

Gene therapy helps combat some forms of blindness – and ongoing clinical trials are looking to extend these treatments to other diseases

An estimated 295 million people suffer from visual impairment globally. Around 43 million of those people are living with blindness. While not every form of blindness can be cured, recent scientific breakthroughs have uncovered new ways to treat some forms of inherited blindness through gene therapy.

Daily Mirror

'People say we are playing God changing our daughter's genetics - but it saved her'

Nicola Kelleher's partner Bradley Benjamin has a hereditary kidney disease which has seen him suffer on dialysis for years - and they didn't want the same outcome for their child

WEKU

Gene therapy for muscular dystrophy stirs hopes and controversy

The FDA is considering greenlighting the experimental treatment under its accelerated approvals program. Some critics point out the therapy isn't yet proven to work and may be costly.

Dynamite News

Genetic therapy can lower tau protein which causes Alzheimer's disease: Study

A world-first trial at UCL and UCLH has found a new gene therapy for Alzheimer's disease that is able to safely lower levels of the harmful tau protein known to cause the disease. Read further on Dynamite News:

LiveScience

Gene therapy: Everything you need to know about the DNA-tweaking treatments

Gene therapies treat or prevent disease by tweaking the content or expression of cells' DNA.

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