#gene-therapy
#gene-therapy

3 days ago

New Jersey teen pain-free thanks to new sickle cell disease treatment

A groundbreaking gene therapy offers a potential cure for sickle cell disease, marking a significant advancement in treatment.

fromArs Technica

The remarkable timeline of a custom gene-editing therapy to save a newborn

KJ received groundbreaking gene-editing therapy at just six months old, showcasing rapid advancements in clinical trial processes.

Venture

fromMedCity News

Cash-Strapped Gene Therapy Firm Bluebird Bio Agrees to Acquisition by Private Equity - MedCity News

Bluebird Bio has agreed to sell itself to private equity firms Carlyle and SK Capital for $29 million to stabilize its gene therapy commercialization efforts.

EU data protection

fromwww.mcall.com

Patient dies following muscular dystrophy gene therapy, Sarepta reports

A patient died from acute liver injury while using Sarepta's Elevidys gene therapy for muscular dystrophy, causing a major stock market reaction.

3 days ago

New Jersey teen pain-free thanks to new sickle cell disease treatment

A groundbreaking gene therapy offers a potential cure for sickle cell disease, marking a significant advancement in treatment.

fromArs Technica

The remarkable timeline of a custom gene-editing therapy to save a newborn

KJ received groundbreaking gene-editing therapy at just six months old, showcasing rapid advancements in clinical trial processes.

Venture

fromMedCity News

Cash-Strapped Gene Therapy Firm Bluebird Bio Agrees to Acquisition by Private Equity - MedCity News

Bluebird Bio has agreed to sell itself to private equity firms Carlyle and SK Capital for $29 million to stabilize its gene therapy commercialization efforts.

EU data protection

fromwww.mcall.com

Patient dies following muscular dystrophy gene therapy, Sarepta reports

A patient died from acute liver injury while using Sarepta's Elevidys gene therapy for muscular dystrophy, causing a major stock market reaction.

more#fda-approval

#personalized-medicine

fromwww.nature.com

In World First, Baby Receives Personalized CRISPR Gene-Editing Treatment

A baby boy is thriving after receiving a personalized CRISPR therapy designed for his unique genetic mutation.

NYC parents

A Baby Received a Custom Crispr Treatment in Record Time

CRISPR technology provides a personalized treatment option for patients with life-threatening genetic disorders.

fromtime.com

A Baby Receives the First Customized CRISPR Treatment

CRISPR technology shows potential in customized gene therapy for rare diseases, specifically in treating a patient with liver mutations.

6 days ago

Carl H. June, creator of CAR-T therapies: The genetic medicine of the future will be able to treat everything, from diabetes to chronic infections'

Carl H. June envisions a future in medicine led by personalized gene therapies that can cure numerous diseases, including cancers and chronic conditions.

World's first personalized CRISPR therapy given to baby with genetic disease

CRISPR therapy shows promise for treating ultra-rare genetic diseases, demonstrated by KJ Muldoon, the first recipient of tailored gene-editing treatment.

fromwww.nature.com

In World First, Baby Receives Personalized CRISPR Gene-Editing Treatment

A baby boy is thriving after receiving a personalized CRISPR therapy designed for his unique genetic mutation.

NYC parents

A Baby Received a Custom Crispr Treatment in Record Time

CRISPR technology provides a personalized treatment option for patients with life-threatening genetic disorders.

fromtime.com

A Baby Receives the First Customized CRISPR Treatment

CRISPR technology shows potential in customized gene therapy for rare diseases, specifically in treating a patient with liver mutations.

6 days ago

Carl H. June, creator of CAR-T therapies: The genetic medicine of the future will be able to treat everything, from diabetes to chronic infections'

Carl H. June envisions a future in medicine led by personalized gene therapies that can cure numerous diseases, including cancers and chronic conditions.

more#personalized-medicine

World's first personalized CRISPR therapy given to baby with genetic disease

CRISPR therapy shows promise for treating ultra-rare genetic diseases, demonstrated by KJ Muldoon, the first recipient of tailored gene-editing treatment.

In a World First, CRISPR Drug Tailored for One Baby Shows Life-Saving Promise

CRISPR technology successfully healed a newborn with a rare genetic disorder, indicating a breakthrough in personalized gene therapy for genetic diseases.

4 months ago

Correcting Genetic Spelling Errors With Next-Generation Crispr

Sam Berns' life epitomizes the urgent need for advancements in genetic treatments, particularly for disorders like progeria that code for rapid aging.

OMG science

Powerful CRISPR system inserts whole gene into human DNA

fromFuturism

In a World First, CRISPR Drug Tailored for One Baby Shows Life-Saving Promise

CRISPR technology successfully healed a newborn with a rare genetic disorder, indicating a breakthrough in personalized gene therapy for genetic diseases.

4 months ago

Correcting Genetic Spelling Errors With Next-Generation Crispr

Sam Berns' life epitomizes the urgent need for advancements in genetic treatments, particularly for disorders like progeria that code for rapid aging.

OMG science

Powerful CRISPR system inserts whole gene into human DNA

more#crispr

#medical-research

3 weeks ago

A Spanish therapy saves the lives of the unluckiest family in the world'

A revolutionary gene therapy has transformed the lives of children suffering from a rare genetic disorder caused by a DNA mutation.

High blood pressure medication for adults also helps children with butterfly skin

New gene therapy shows promise in healing epidermolysis bullosa wounds, while repurposed drugs like losartan also provide potential benefits.

OMG science

New research shows promise for restoring vision for people with glaucoma, other conditions

3 weeks ago

A Spanish therapy saves the lives of the unluckiest family in the world'

A revolutionary gene therapy has transformed the lives of children suffering from a rare genetic disorder caused by a DNA mutation.

High blood pressure medication for adults also helps children with butterfly skin

New gene therapy shows promise in healing epidermolysis bullosa wounds, while repurposed drugs like losartan also provide potential benefits.

OMG science

New research shows promise for restoring vision for people with glaucoma, other conditions

more#medical-research

3 weeks ago

Using brain-dead people for medical experiments: The new debate at the frontier of bioethics

Using brain-dead bodies for medical experiments could significantly advance research into lethal diseases.

Long Island patient gets breakthrough sickle cell treatment called a 'cure': 'I'm not in pain anymore'

Lyfgenia gene therapy successfully cured sickle cell disease in a New Yorker, showcasing a major medical breakthrough.

Healthcare

fromFast Company

These breakthrough gene therapies cost millions of dollars. Is a mortgage model the secret to paying for them?

Insurers' refusal to cover innovative gene therapies for conditions like sickle cell disease jeopardizes patient access, necessitating creative financing solutions.

NYC parents

fromNew York Post

Long Island patient gets breakthrough sickle cell treatment called a 'cure': 'I'm not in pain anymore'

Lyfgenia gene therapy successfully cured sickle cell disease in a New Yorker, showcasing a major medical breakthrough.

Healthcare

fromFast Company

These breakthrough gene therapies cost millions of dollars. Is a mortgage model the secret to paying for them?

Insurers' refusal to cover innovative gene therapies for conditions like sickle cell disease jeopardizes patient access, necessitating creative financing solutions.

more#sickle-cell-disease

#health-innovation

fromwww.dailymail.co.uk

Inside the secret island where wealthy people go to alter their DNA

Roatan is attracting ultra-wealthy individuals seeking experimental gene therapies due to its lack of regulations around such treatments.

fromEntrepreneur

1 month ago

This Gene Therapy Startup Wants to Change the Way We Age | Entrepreneur

Minicircle aims to slow aging and enhance biological function through a quick gene therapy injection.

fromwww.dailymail.co.uk

Inside the secret island where wealthy people go to alter their DNA

Roatan is attracting ultra-wealthy individuals seeking experimental gene therapies due to its lack of regulations around such treatments.

fromEntrepreneur

1 month ago

This Gene Therapy Startup Wants to Change the Way We Age | Entrepreneur

Minicircle aims to slow aging and enhance biological function through a quick gene therapy injection.

more#health-innovation

Gene therapy frees hemophilia patient from constant worry, needles - Harvard Gazette

Gene therapy is providing hope and improved quality of life for hemophilia patients.

Miscellaneous

The second revolution in gene therapy that has cured Javier, a child with a lethal error in the X chromosome

A small sign led to diagnosis of Wiskott-Aldrich syndrome, which was overcome through groundbreaking gene therapy.

fromHarvard Gazette

1 month ago

Gene therapy frees hemophilia patient from constant worry, needles - Harvard Gazette

Gene therapy is providing hope and improved quality of life for hemophilia patients.

Miscellaneous

The second revolution in gene therapy that has cured Javier, a child with a lethal error in the X chromosome

A small sign led to diagnosis of Wiskott-Aldrich syndrome, which was overcome through groundbreaking gene therapy.

Northern Minnesota boy is first American to receive groundbreaking treatment for muscular dystrophy

Colton Belluzzo received groundbreaking gene therapy for Duchenne muscular dystrophy, showing promise in slowing disease progression.

fromNews Center

Uncovering New Therapeutic Targets for Pediatric Epilepsy - News Center

Researchers have identified new mechanisms in Dravet syndrome that highlight novel therapeutic targets, particularly in intronic regions of the SCN1A gene.

Gene therapy experiment gives children 'life-changing' sight boost

Gene therapy has significantly improved the vision of toddlers born with severe childhood blindness, providing life-changing outcomes.

London startup

fromwww.theguardian.com

Doctors in London cure blindness in children with rare condition

Doctors in London have pioneered gene therapy to cure childhood blindness caused by a genetic condition, marking a significant advancement in medical science.

Medicine

fromwww.nytimes.com

Mutated DNA Restored to Normal in Gene Therapy Advance

A single infusion successfully corrected a mutated gene, marking a significant advancement in gene therapy.

This precision targeting could revolutionize treatment for various genetic diseases.

fromwww.bbc.com

Gene therapy experiment gives children 'life-changing' sight boost

Gene therapy has significantly improved the vision of toddlers born with severe childhood blindness, providing life-changing outcomes.

London startup

fromwww.theguardian.com

Doctors in London cure blindness in children with rare condition

Doctors in London have pioneered gene therapy to cure childhood blindness caused by a genetic condition, marking a significant advancement in medical science.

Medicine

fromwww.nytimes.com

Mutated DNA Restored to Normal in Gene Therapy Advance

A single infusion successfully corrected a mutated gene, marking a significant advancement in gene therapy.

This precision targeting could revolutionize treatment for various genetic diseases.

more#medical-innovation

Mental health

Why women's brains are more resilient: it could be their 'silent' X chromosome

The second X chromosome in females may guard against cognitive decline and aging.

Wearables

The search for solutions to geographic atrophy of the retina

Age-related macular degeneration (AMD) leads to progressive vision loss, significantly impacting independence, but recent drug approvals offer hope for treatment.

fromwww.standard.co.uk

Gene therapy experiment gives children 'life-changing' sight boost at London hospital

Pioneering gene therapy restores sight in young children with a rare genetic condition.