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41 Resultados de su búsqueda "Gene Therapy".

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15 Oct
Gene Therapy Might Tweak Fats, Help Prevent Arthritis in Overweight Kids

Gene Therapy Might Tweak Fats, Help Prevent Arthritis in Overweight Kids

Gene therapy could help obese kids avoid health problems associated with excess weight, including arthritis, a new, preliminary study in mice suggests.

The gene therapy prompts...

Gene TherapyArthritis: Misc.
10 Oct
Boys Treated With Gene Therapy for Rare Brain Disease Doing Well 6 Years Later

Boys Treated With Gene Therapy for Rare Brain Disease Doing Well 6 Years Later

Most boys treated with breakthrough gene therapy for a rare but deadly brain illness are faring well six years later, two new reports find.

The 77 boys were treated for cerebral adrenoleukodystrophy (CALD), a formerly incurable and progressive genetic brain disease that ...

BrainGene Therapy
26 Sep
Trial Confirms 'Life-Changing' Impact of Gene Therapy for Hemophilia B

Trial Confirms 'Life-Changing' Impact of Gene Therapy for Hemophilia B

A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows.

People with hemophilia B saw their bleeding episodes drop by an average 71% following a single infusion...

Gene TherapyHemophilia
06 Sep
Gene Therapy Reverses a Rare Cause of Vision Loss

Gene Therapy Reverses a Rare Cause of Vision Loss

Gene therapy may restore vision to children and adults robbed of their sight by a rare inherited condition called Leber congenital amaurosis, researchers report.

The illness is caused by mutations in the GUCY2D gene, which is critical to producing proteins that enable vi...

Eye / Vision Problems: Misc.Gene Therapy
18 Jun
Another Study Finds CRISPR Gene Therapy Fights Sickle Cell

Another Study Finds CRISPR Gene Therapy Fights Sickle Cell

There's more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patien...

Gene TherapySickle-Cell Anemia
05 Jun
Gene Therapy in Both Ears Restores Hearing to Babies Born Deaf

Gene Therapy in Both Ears Restores Hearing to Babies Born Deaf

Chlidren born deaf have had their hearing restored in both ears as a result of gene therapy, a new study reports.

All five children showed hearing recovery in both ears, with dramatic improvements in speech perception and the ability to locate the position of a sound.

Gene TherapyHearing Loss
15 May
Early Hints at a Gene Therapy Cure for Herpes

Early Hints at a Gene Therapy Cure for Herpes

An experimental gene therapy could one day provide a first-ever cure for genital and oral herpes, researchers report.

The gen...

Gene TherapySexually Transmitted Diseases: Herpes, Genital
10 May
Baby Girl Born Deaf Gains Hearing After Gene Therapy

Baby Girl Born Deaf Gains Hearing After Gene Therapy

Opal Sandy was born into a world she could not hear. 

The British baby girl, now 18 months old, had a rare genetic condition called auditory neuropathy that interrupted nerve impulses that travel from the inner ear to the brain. She'd been fitted with a cochlear imp...

Gene Therapy
06 May
Gene Therapy Improves Vision in People With Inherited Blindness

Gene Therapy Improves Vision in People With Inherited Blindness

An injectable gene therapy caused measurable improvements in vision among a small group of people with inherited blindness, an early-stage clinical trial says.

Researchers recruited 14 people with Leber Congenital Amaurosis (LCA), a rare genetic condition that causes bab...

Eye / Vision Problems: Misc.Gene Therapy
25 Jan
Gene Therapy Brings Hearing to Kids With Congenital Deafness

Gene Therapy Brings Hearing to Kids With Congenital Deafness

Five of six Chinese children born deaf due to a rare genetic defect now have the ability to hear, thanks to an experimental gene therapy.

The therapy involved a hollowed-out virus loaded with a healthy version of the gene responsible for producing otoferlin, a protein ne...

Gene TherapyHearing Loss
08 Dec
FDA Approves Landmark Sickle Cell Gene Therapies

FDA Approves Landmark Sickle Cell Gene Therapies

The U.S. Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology.

Casgevy, developed by Vertex Pharmaceuticals of Boston and CRISPR Therapeutics of...

Blood DisordersGene TherapySickle-Cell Anemia
13 Nov
Two New Studies Point to the Promise of Gene Therapy for High Cholesterol

Two New Studies Point to the Promise of Gene Therapy for High Cholesterol

MONDAY, Nov. 13, 2023 (Healthday News) -- Two new gene-editing treatments that target dangerously high levels of cholesterol in people with a genetic predisposition to the condition were found safe and effective in new, groundbreaking research.

While powerful drugs like ...

GeneticsCholesterol: DietaryGene TherapyHeart / Stroke-Related: High Cholesterol
27 Sep
In Mice, Gene Therapy Helps Restore Movement After Spinal Cord Injury

In Mice, Gene Therapy Helps Restore Movement After Spinal Cord Injury

Gene therapy has restored mobility in mice with completely severed spinal cords, researchers report.

The mice regained the ability to walk, with gait patterns resembling those of mice that resumed walking naturally after only partial cord injuries, the investigators foun...

Gene TherapySpinal Problems
25 Sep
Surgeons Perform Transplant of Gene-Tweaked Pig Heart Into Second Patient

Surgeons Perform Transplant of Gene-Tweaked Pig Heart Into Second Patient

A second human patient has received a genetically altered pig heart as he battles the ravages of end-stage heart disease.

The 58-year-old man, Lawrence Faucette, received the pig organ at the University of Maryland Medical Center in Baltimore.

The medical team was ...

Surgery: Misc.Gene TherapyOrgan Transplants
31 Aug
Gene-Tweaked Stem Cells Offer Hope Against Sickle Cell Disease

Gene-Tweaked Stem Cells Offer Hope Against Sickle Cell Disease

A type of gene therapy that precisely "edits" a key bit of DNA might offer a new way to treat sickle cell disease -- a painful inherited condition that largely strikes Black children and adults.

That's according to a new study in the

Sickle-Cell AnemiaGene Therapy
14 Aug
In Monkey Trial, Gene Therapy Shows Promise in Curbing Severe Problem Drinking

In Monkey Trial, Gene Therapy Shows Promise in Curbing Severe Problem Drinking

For people with severe alcohol use disorder, a new gene therapy trial could lead to an effective treatment that would involve chemically rebalancing the area of the brain associated with addiction.

"With alcohol alone, there's generally more than 100,000 deaths [in the ...

Gene TherapyAddictionAlcohol Abuse
17 Jul
Gene-Editing Tools Pave Way for New Alzheimer's Treatments

Gene-Editing Tools Pave Way for New Alzheimer's Treatments

Two new studies using CRISPR gene editing offer potential new treatments for Alzheimer's disease.

"A pipeline of potential new treatments offers hope for the Alzheimer's and dementia community,"said

Alzheimer'sGene TherapyDementia
12 Jun
More Evidence Gene Therapy Might Cure Sickle Cell Disease

More Evidence Gene Therapy Might Cure Sickle Cell Disease

A clinical trial that's attempting to discover a cure for sickle cell disease has found a new gene therapy to be safe and successful in four patients.

Two of the patients were treated at Cleveland Clinic Children's in Ohio, and doctors there are hopeful that their positi...

Sickle-Cell AnemiaGene TherapyStem CellsClinical Trials
06 Jun
Injected Birth Control Could Be Game-Changer to Curb Stray Cat Populations

Injected Birth Control Could Be Game-Changer to Curb Stray Cat Populations

Millions of stray cats roam the world over, and surgical sterilization has long been the primary method of population control.

But a small new study shows promising results for a one-and-done contraceptive injection.

Researchers say this first-of-its-kind appr...

Hormones: Misc.EnvironmentBirth ControlGene TherapyPets And Health
31 May
Sickle Cell Gene Therapy Can Cure, But Costs Almost $3 Million. Who Will Pay?

Sickle Cell Gene Therapy Can Cure, But Costs Almost $3 Million. Who Will Pay?

An exceptionally pricey gene therapy cure for sickle cell disease could soon be available, but it's not clear whether insurance companies will balk at the cost and deny coverage.

On the surface, the gene therapy does not appear as cost-effective as the grinding medical c...

Sickle-Cell AnemiaHealth Care Access / DisparitiesHealth CostsGene Therapy
26 Jan
New Breakthrough Could Speed Gene Therapy Research

New Breakthrough Could Speed Gene Therapy Research

"Zinc fingers"might sound like the world's worst candy bar, but these human proteins might prove key to treating complex genetically driven diseases.

A new artificial intelligence program is poised to enable the simple production of zinc fingers, according to

Gene Therapy
22 Dec
Gene Therapy Breakthrough Helps Children Born Without Immune System

Gene Therapy Breakthrough Helps Children Born Without Immune System

Ten children with an especially rare and hard-to-treat form of "bubble boy" disease are living normal lives after receiving a new gene therapy approach, researchers say.

Experts said the findings are a major advance for children with the disease -- a subtype of seve...

Gene TherapyKids: Misc.Immune Disorders
19 Dec
FDA Approves Gene Therapy for Tough-to-Treat Bladder Cancer

FDA Approves Gene Therapy for Tough-to-Treat Bladder Cancer

Patients with a high-risk bladder cancer now have a new option to treat it.

The U.S. Food and Drug Administration on Friday approved a gene therapy called Adstiladrin, which is designed to work for patients who have what's called high-risk non-muscle-invasive bladder can...

Cancer: BladderGene TherapyFood &, Drug Administration
15 Dec
Gene Therapy Gel Offers New Hope Against Rare Blistering Disease

Gene Therapy Gel Offers New Hope Against Rare Blistering Disease

An experimental gene therapy that's applied as a skin gel appears to heal wounds caused by a rare and severe genetic skin disease.

Experts called the findings "remarkable," and said they bring hope of a better quality of life to children and young adults living with the ...

Skin Disorders: Misc.Gene Therapy
23 Nov
A Gene Therapy for Hemophilia That Costs $3.5 Million Gets FDA Approval

A Gene Therapy for Hemophilia That Costs $3.5 Million Gets FDA Approval

People with one form of the genetic blood disorder hemophilia now have a one-time treatment with a $3.5 million price tag.

The U.S. Food and Drug Administration approved the new gene therapy Hemgenix on Nov. 22. Soon after, drugmaker CSL Behring revealed its cost.

Health Care Access / DisparitiesGene TherapyHemophiliaDrug ApprovalsFood &, Drug Administration
10 Nov
Gene Therapy Used for First Time to Correct Fatal Illness Before Birth

Gene Therapy Used for First Time to Correct Fatal Illness Before Birth

Doctors are hopeful that an innovative treatment performed before birth may help children born with the rare genetic, and often fatal, condition called Pompe disease.

A thriving Canadian toddler is evidence that treatment while still in the womb offers better outcomes.

Gene TherapyGenetic DisordersInfant / Child Care
10 Nov
Doctors Use CRISPR Technology to Boost Cancer Immunotherapy

Doctors Use CRISPR Technology to Boost Cancer Immunotherapy

Gene editing has for the first time produced modified immune cells finely honed to target and attack cancer cells, researchers say.

A team used the gene editing tool CRISPR to alter immune cells drawn from 16 patients who had a variety of solid cancers, including colon, ...

BiotechnologyGene TherapyGenetics
13 Oct
Gene Therapy Helps Those With Rare Form of Blindness See Better at Night

Gene Therapy Helps Those With Rare Form of Blindness See Better at Night

An experimental gene therapy offers hope for rapid improvement in the night vision of adults who have a congenital form of childhood-onset blindness, researchers report.

In this ongoing...

Eye / Vision Problems: Misc.Gene TherapyBlindness
22 Jul
Gene Therapy Makes Inroads Against a Form of Hemophilia

Gene Therapy Makes Inroads Against a Form of Hemophilia

People with hemophilia B could find their bleeding risk dramatically reduced with just one injection of an experimental gene therapy, a new study reports.

Hemophilia B is a rare and inherited ...

Gene TherapyHemophilia
13 May
In Animal Studies, Gene Therapy Shows Promise for Spinal Cord Injury Pain

In Animal Studies, Gene Therapy Shows Promise for Spinal Cord Injury Pain

An experimental gene therapy for spinal cord pain shows promise in mice, researchers say.

About half of spinal cord injury patients have neuropathy, which is chronic or debilitating pain, tingling, numbn...

PainGene TherapySpinal Problems
29 Mar
Saving the 'Butterfly Children:' Gene Therapy Helps Heal Deadly Blistering Condition

Saving the 'Butterfly Children:' Gene Therapy Helps Heal Deadly Blistering Condition

An experimental cream-based gene therapy may soon become the first U.S. government-approved means for treating a rare and devastating skin disease that produces "butterfly children."

Patients with recessive dystrophic

Skin Disorders: Misc.Clinical TrialsGene TherapyGenetics
25 Feb
Researchers May Be Close to a Cure for Type 1 Diabetes

Researchers May Be Close to a Cure for Type 1 Diabetes

Science could be well on its way to a cure for type 1 diabetes, as researchers hone transplant therapies designed to restore patients' ability to produce t...

Gene TherapyStem CellsDiabetes: Type I
16 Feb
Gene Editing on Ticks Promises Insights Into Disease Prevention

Gene Editing on Ticks Promises Insights Into Disease Prevention

Scientists who conducted the first gene editing in ticks say this line of research could lead to new ways to reduce tick-borne diseases in humans.

Ticks can transmit a wide number of diseases to ...

Lyme DiseaseGene TherapyInfections: Misc.
08 Feb
Acne's Genetic Secrets Could Bring Better Treatments

Acne's Genetic Secrets Could Bring Better Treatments

For countless teens, it's the scourge of adolescence. But researchers say the discovery of new genetic variants associated with acne could help doctors identify people at high risk and perhaps point the way to new treatments.

"Despite major treatment advances in other sk...

Skin Disorders: Misc.Gene TherapyAcne
02 Feb
Ten Years After Gene Therapy for Leukemia, Doctors Say Patients Cured

Ten Years After Gene Therapy for Leukemia, Doctors Say Patients Cured

More than 10 years after Doug Olsen underwent an experimental gene therapy that turned his T-cells into cancer killers, his leukemia

Gene TherapyCancer: LeukemiaCancer: Lymphoma
13 Dec
Could Gene Therapy Help Cure Sickle Cell Disease?

Could Gene Therapy Help Cure Sickle Cell Disease?

A gene therapy that could provide a permanent cure for sickle cell disease continues to show success through a third wave of patients, researchers report.

The therapy, LentiGlobin, restored normal blood function in 35 sickle cell patients who had the one-time procedure, ...

Blood DisordersCancer: LeukemiaClinical TrialsGene TherapyGenetic DisordersResearch &, DevelopmentSickle-Cell AnemiaStem CellsTherapy &, Procedures: Misc.
18 Nov
Gene Therapy Could Be Big Advance Against Hemophilia

Gene Therapy Could Be Big Advance Against Hemophilia

Gene therapy shows promise in reducing, and even halting, potentially life-threatening bleeding events in people with hemophilia, researchers report.

Hemophilia A is the most common inherited bleeding disorder, affecting one in 5,000 males worldwide. It's caused by a mis...

Blood DisordersGene TherapyHemophiliaResearch &, DevelopmentTherapy &, Procedures: Misc.
18 Nov
Gene Therapy May Reverse Hurler Syndrome, a Rare and Severe Illness in Kids

Gene Therapy May Reverse Hurler Syndrome, a Rare and Severe Illness in Kids

Gene therapy might soon offer a new option for children with a rare genetic disorder that damages tissues throughout the body, researchers are reporting.

In a study of eight children with the condition, called Hurler syndrome, researchers found that the gene therapy was ...

Birth Defects: Misc.Child DevelopmentGene TherapyGenetic DisordersKids' AilmentsStem CellsTherapy &, Procedures: Misc.
04 Nov
Mouse Study Offers Hope for Gene Therapy Against Parkinson's Disease

Mouse Study Offers Hope for Gene Therapy Against Parkinson's Disease

An experimental gene therapy to boost the effectiveness of the Parkinson's drug levodopa yielded promising results in mice, researchers report.

As the loss of dopamine-releasing neurons advances in late-stage Parkinson's, levodopa is less able to ease movement problems c...

BrainGene TherapyParkinson's
21 Oct
Ten Years On, Gene Therapy Still Beating Most Cases of 'Bubble Boy' Immune Disease

Ten Years On, Gene Therapy Still Beating Most Cases of 'Bubble Boy' Immune Disease

Nine of 10 patients with so-called "bubble boy" immune disease who received gene therapy about a decade ago are still disease-free, researchers report.

The gene therapy was developed at the University of California, Los Angeles (UCLA), to treat the rare and deadly immune...

Gene TherapyGenetic DisordersImmune DisordersInfections: Misc.Research &, DevelopmentStem CellsTherapy &, Procedures: Misc.
16 Sep
Even When Undergoing Treatment, People With MS Gain From COVID Vaccines

Even When Undergoing Treatment, People With MS Gain From COVID Vaccines

Multiple sclerosis (MS) patients undergoing a treatment that depletes a type of immune cell that fuels MS attacks still have a strong response to mRNA COVID-19 vaccines, a new study finds.

"The message from this study is clear -- it is worthwhile for patients with MS rec...

Gene TherapyImmunizationInfections: Misc.Multiple SclerosisSafety &, Public HealthVaccines

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