#gene-therapy

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fromNature
4 days ago

Brain editing now 'closer to reality': the gene-altering tools tackling deadly disorders

Researchers are advancing genome editing techniques to target brain disorders, aiming for human trials within a few years.
#fda
fromwww.nature.com
1 week ago
Health

Therapeutic genetic restoration through allogeneic brain microglia replacement

A brain-restricted microglia replacement approach avoids myeloablation and enhances therapy for lysosomal storage diseases.
Science
fromNature
3 weeks ago

Made-to-order DNA goes big: new tech doubles size of custom genetic sequences

Synthetic biology solutions require innovative DNA synthesis methods for commercial viability.
fromwww.npr.org
1 month ago

She hoped key research could help save her eyesight. Then the Trump funding cuts came

Cuts to federal research funding threaten advancements in gene therapies crucial for managing vision loss in individuals with Usher syndrome.
fromNature
2 months ago

One dose of gene therapy gives years of relief from blood disorder

Gene therapy for hemophilia B significantly reduces bleeding episodes and shows long-term effectiveness.
fromwww.theguardian.com
2 months ago

Transformative': the UK lab working on a way to halt genetic type of dementia

"It may be one of the first dementias to have a definitive treatment, a cure if you like, a really transformative treatment that allows people to live much longer and much more normal lives..."
London startup
#neurodegenerative-diseases
NYC startup
fromNews Center
2 months ago

Taking the Science to Market: Start-ups at Feinberg - News Center

Significant discoveries in lab research can lead to the creation of start-ups focused on commercializing innovative medical treatments.
NYC startup
fromNews Center
2 months ago

Taking the Science to Market: Start-ups at Feinberg - News Center

Significant discoveries in lab research can lead to the creation of start-ups focused on commercializing innovative medical treatments.
#crispr
fromTODAY.com
2 months ago
Parenting

EXCLUSIVE: Baby KJ, Saved by New Gene Editing Therapy, Goes Home After 300 Days in Hospital

Parenting
fromNature
3 months ago

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.
Alternative medicine
fromtime.com
3 months ago

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.
Parenting
fromTODAY.com
2 months ago

EXCLUSIVE: Baby KJ, Saved by New Gene Editing Therapy, Goes Home After 300 Days in Hospital

KJ Muldoon is the first patient treated with customized CRISPR gene therapy, successfully overcoming a rare genetic disorder.
Growth hacking
fromFuturism
3 months ago

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.
NYC parents
fromWIRED
3 months ago

A Baby Received a Custom Crispr Treatment in Record Time

CRISPR technology provides a personalized treatment option for patients with life-threatening genetic disorders.
Parenting
fromNature
3 months ago

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.
Alternative medicine
fromtime.com
3 months ago

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.
SF parents
fromCbsnews
2 months 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.
fromenglish.elpais.com
2 months 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'

The transformation in medicine driven by personalized gene therapies promises cures for many diseases, including cancer, diabetes, and autoimmune conditions, but widespread application may take decades.
Alternative medicine
Parenting
fromArs Technica
3 months ago

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.
fromNature
3 months ago

Powerful CRISPR system inserts whole gene into human DNA

CRISPR offers more control than viral carriers do, but typically requires the cutting of DNA - raising the chances of unwanted mutations and incomplete repair.
OMG science
Alternative medicine
fromenglish.elpais.com
3 months ago

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.
SF parents
fromenglish.elpais.com
3 months 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.
OMG science
fromenglish.elpais.com
3 months 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.
Healthcare
fromFast Company
3 months ago

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.
fromCbsnews
3 months ago

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

New research reveals potential therapies for retinal damage using gene therapy to combat Prox-1 protein's inhibitory effect on retinal regeneration.
Growth hacking
fromEntrepreneur
3 months 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.
OMG science
fromHarvard Gazette
4 months 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.
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