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Health News Results - 47
Gene Therapy Brings Hearing to Kids With Congenital Deafness
- Dennis Thompson HealthDay Reporter
- January 25, 2024
- Full Page
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...
FDA Approves Landmark Sickle Cell Gene Therapies
- Dennis Thompson HealthDay Reporter
- December 8, 2023
- Full Page
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...
Two New Studies Point to the Promise of Gene Therapy for High Cholesterol
- Robin Foster HealthDay Reporter
- November 13, 2023
- Full Page
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 ...
In Mice, Gene Therapy Helps Restore Movement After Spinal Cord Injury
- Dennis Thompson HealthDay Reporter
- September 27, 2023
- Full Page
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...
Surgeons Perform Transplant of Gene-Tweaked Pig Heart Into Second Patient
- Cara Murez HealthDay Reporter
- September 25, 2023
- Full Page
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 ...
Gene-Tweaked Stem Cells Offer Hope Against Sickle Cell Disease
- Amy Norton HealthDay Reporter
- August 31, 2023
- Full Page
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.
In Monkey Trial, Gene Therapy Shows Promise in Curbing Severe Problem Drinking
- Sarah D. Collins HealthDay Reporter
- August 14, 2023
- Full Page
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 th...
Gene-Editing Tools Pave Way for New Alzheimer's Treatments
- Cara Murez HealthDay Reporter
- July 17, 2023
- Full Page
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
More Evidence Gene Therapy Might Cure Sickle Cell Disease
- Cara Murez HealthDay Reporter
- June 12, 2023
- Full Page
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...
Injected Birth Control Could Be Game-Changer to Curb Stray Cat Populations
- Alan Mozes HealthDay Reporter
- June 6, 2023
- Full Page
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...
Sickle Cell Gene Therapy Can Cure, But Costs Almost $3 Million. Who Will Pay?
- Dennis Thompson HealthDay Reporter
- May 31, 2023
- Full Page
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...
New Breakthrough Could Speed Gene Therapy Research
- Dennis Thompson HealthDay Reporter
- January 26, 2023
- Full Page
“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 Breakthrough Helps Children Born Without Immune System
- Amy Norton HealthDay Reporter
- December 22, 2022
- Full Page
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...
FDA Approves Gene Therapy for Tough-to-Treat Bladder Cancer
- Cara Murez HealthDay Reporter
- December 19, 2022
- Full Page
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...
Gene Therapy Gel Offers New Hope Against Rare Blistering Disease
- Amy Norton HealthDay Reporter
- December 15, 2022
- Full Page
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 ...
A Gene Therapy for Hemophilia That Costs $3.5 Million Gets FDA Approval
- Cara Murez HealthDay Reporter
- November 23, 2022
- Full Page
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.
Gene Therapy Used for First Time to Correct Fatal Illness Before Birth
- Cara Murez HealthDay Reporter
- November 10, 2022
- Full Page
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.
Doctors Use CRISPR Technology to Boost Cancer Immunotherapy
- Dennis Thompson HealthDay Reporter
- November 10, 2022
- Full Page
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, ...
Gene Therapy Helps Those With Rare Form of Blindness See Better at Night
- By Cara Murez HealthDay Reporter
- October 13, 2022
- Full Page
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...
Gene Therapy Makes Inroads Against a Form of Hemophilia
- By Dennis Thompson HealthDay Reporter
- July 22, 2022
- Full Page
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 ...
In Animal Studies, Gene Therapy Shows Promise for Spinal Cord Injury Pain
- By Robert Preidt HealthDay Reporter
- May 13, 2022
- Full Page
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...
Saving the 'Butterfly Children:' Gene Therapy Helps Heal Deadly Blistering Condition
- Dennis Thompson
- March 29, 2022
- Full Page
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."
Researchers May Be Close to a Cure for Type 1 Diabetes
- February 25, 2022
- Full Page
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 Editing on Ticks Promises Insights Into Disease Prevention
- February 16, 2022
- Full Page
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.
Acne's Genetic Secrets Could Bring Better Treatments
- February 8, 2022
- Full Page
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...
Ten Years After Gene Therapy for Leukemia, Doctors Say Patients Cured
- Robin Foster and Robert Preidt
- February 2, 2022
- Full Page
More than 10 years after Doug Olsen underwent an experimental gene therapy that turned his T-cells into cancer killers, his leukemia
Could Gene Therapy Help Cure Sickle Cell Disease?
- Dennis Thompson HealthDay Reporter
- December 13, 2021
- Full Page
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, ...
Gene Therapy Could Be Big Advance Against Hemophilia
- Robert Preidt
- November 18, 2021
- Full Page
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...
Gene Therapy May Reverse Hurler Syndrome, a Rare and Severe Illness in Kids
- Amy Norton HealthDay Reporter
- November 18, 2021
- Full Page
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 ...
Mouse Study Offers Hope for Gene Therapy Against Parkinson's Disease
- Robert Preidt
- November 4, 2021
- Full Page
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...
Ten Years On, Gene Therapy Still Beating Most Cases of 'Bubble Boy' Immune Disease
- Robert Preidt
- October 21, 2021
- Full Page
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...
Even When Undergoing Treatment, People With MS Gain From COVID Vaccines
- Steven Reinberg
- September 16, 2021
- Full Page
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...
Pig Study Could Lead to Gene Therapy to Prevent Heart Failure
- Amy Norton HealthDay Reporter
- July 1, 2021
- Full Page
A gene therapy aimed at freeing the heart's capacity for self-repair has shown early promise in an animal study.
The study -- done in pigs -- found that the treatment approach was not only feasible, but also improved the animals' heart function after they sustained ...
Man Blind for 40 Years Regains Some Sight Through Gene Therapy
- May 24, 2021
- Full Page
Doctors for the first time have used a form of gene therapy to restore partial vision in a blind person, according to findings announced Monday.
The research team genetically altered retinal ganglion cells to become light-sensitive in a man whose vision was destroyed by ...
Gene Therapy Uses HIV to Rescue Kids Born Without Immune System
- Dennis Thompson HealthDay Reporter
- May 12, 2021
- Full Page
Cora Oakley is a rough-and-tumble 4-year-old who loves gymnastics and outdoor activities, particularly if it involves bouncing on a trampoline.
It's hard to tell from looking at her that she was born without an immune system. Kids with this condition can acquire dangerou...
Early Promise for Therapy Against Duchenne Muscular Dystrophy
- Robert Preidt
- January 28, 2021
- Full Page
An experimental gene therapy for Duchenne muscular dystrophy shows promise, a small study suggests.
The severe form of muscular dystrophy -- which affects about one in 3,500 males born each year in the United States -- causes muscles to progressively weaken and lose the ...
Could Gene Therapy Cure Sickle Cell Disease? Two New Studies Raise Hopes
- Ernie Mundell
- December 5, 2020
- Full Page
A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients' bone marrow cells, researchers report.
Both therapies work by switching on a gene that promotes production of fetal hemogl...
Could Gene Therapy Stem the Damage of Parkinson's?
- Robert Preidt
- August 19, 2020
- Full Page
It may be possible to protect Parkinson's patients' brains from further damage by turning off a "master regulator" gene, researchers report.
"One of the biggest challenges in treating Parkinson's, other than the lack of therapies that impede disease progression, is t...
CRISPR Gene Editing Creates 'Designer' Immune Cells That Fight Cancer
- Amy Norton
- February 6, 2020
- Full Page
In a first, scientists have used gene-editing technology to create "designer" immune system cells that can fight tumors and survive for months in cancer patients' bodies.
It's a proof of principle, the researchers say -- and an early step toward bringing the gene-edi...
Researchers Alter Mosquitoes to Resist Dengue Infection
- Robert Preidt
- January 16, 2020
- Full Page
Mosquitoes that can't be infected by or spread dengue virus have been created by scientists.
The researchers genetically engineered the mosquitoes to be resistant to all four types of dengue, a mosquito-borne virus that's a significant global health threat.
Gene Therapy May Be Long-Term Cure for Type of Hemophilia
- Dennis Thompson
- January 2, 2020
- Full Page
A new gene therapy appears to serve as a functional cure for the most common type of hemophilia, early clinical trial results indicate.
Patients who received the one-time intravenous therapy continue to have a more than 90% decrease in bleeding events two to three ...
'Designer Babies' a Long Way Off
- Robert Preidt
- November 21, 2019
- Full Page
"Designer babies" aren't going to be a reality anytime soon, researchers say.
Concerns about genetically altering embryos to have desired traits have been around nearly as long as in vitro fertilization (IVF) and the technology to screen embryos have existed.
...
How Chinese Gene-Editing Could Backfire: Babies Might Have Shorter Lives
- Amy Norton
- June 3, 2019
- Full Page
Last year, a scientist from China created a storm of controversy when he claimed he'd used gene-editing technology to create "designer" twin babies. Now, a new study is highlighting one of the dangers of that endeavor.
Researchers have found that the gene mutation th...
Gene Therapy May Help Fight Tough-to-Treat Blood Cancer
- Amy Norton
- May 2, 2019
- Full Page
A gene therapy that tweaks the immune system might offer hope to people with blood cancer that has resisted standard treatments, a new preliminary trial suggests.
The cancer, called multiple myeloma, arises in certain white blood cells. It is currently incurable, but...
Gene Therapy Might Prove a Cure for 'Bubble Boy' Disease
- Dennis Thompson
- April 17, 2019
- Full Page
They were once imprisoned in plastic bubbles that cut them off from the dangerous, infectious world.
But now, children born with a disease that robs them of a functioning immune system may have gained a new lease on life.
Researchers say gene therapy may b...
After Chinese Infant Gene-Editing Scandal, U.S. Health Officials Join Call for a Ban
- Robert Preidt
- March 13, 2019
- Full Page
The controversy over a Chinese scientist who claimed he created gene-edited babies has prompted the U.S. National Institutes of Health to join an international moratorium on such research.
"Today, leading scientists and ethicists from seven countries have called for ...
Someday, a Pig's Heart Might Save a Child's Life
- Amy Norton
- January 28, 2019
- Full Page
The supply of donor organs for infants needing a heart transplant is critically low, but researchers have taken a first step toward using pig hearts to fill the need.
The concept of using animal organs to save human lives has been around for years. With donor organs ...