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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 patients.

“It’s encouraging that this gene-editing treatment continues to show promising efficacy for sickle cell pa...

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.

Two of the children also gained an ability to appreciate music, and were observed dancing to music, researchers adde...

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 gene therapy removed 90% or more of oral herpes infection in lab mice, and it also suppressed how much virus an infected an...

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 implant, but it could only help so much.

Then came a breakthrough gene therapy

  • Ernie Mundell HealthDay Reporter
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  • May 10, 2024
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  • 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 babies to lose some or all of their sight from birth.

    Eleven of the 14 had measurable improvements in the vision of o...

    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 necessary for the transmission of sound signals from the inner ear to the brain.

    Doctors injected the virus into the...

    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 Switzerland, is the first medicine available in the United States to treat a genetic disease using the CRISPR gene-edit...

    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 statins can help manage cholesterol in most people, they can't treat those who have genes that predispose them to heart ...

    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 found.

    This happened because the new gene therapy used techniques to not only repair spinal cord tissue, but also to d...

    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 the same one that performed the first pig transplant with another patient in January 2022.

    “We are once again o...

    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 New England Journal of Medicine desc...

    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 United States] per year,” said

  • Sarah D. Collins HealthDay Reporter
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  • August 14, 2023
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  • 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 Maria Carrillo, chief science officer for the Alzheimer's Association. “The progress and appro...

    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 positive results will be borne out in future research.

    “New treatments like this are critical for people who have sick...

    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 approach appears safe and effective.

    “A non-surgical contraceptive that could result in lifetime sterility following...

    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 care that sickle cell patients now receive, according to a new analysis.

    Gene therapy applied just once to a sickle...

    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 research co-led by ...

    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 severe combined immunodeficiency (SCID).

    SCID refers to a group of rare genetic diseases that cause babies to be born ...

    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 cancer (NMIBC) that hasn't responded to the standard treatment, Bacillus Calmette-Guérin (BCG), but hasn't spread. BCG is ...

    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 condition, called dystrophic epidermolysis bullosa (DEB).

    The disease affects about 3 out of every 1 million peopl...

    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.

    The company said its drug would ultimately reduce health care costs because patients with the genetic disorder would ne...

    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.

    Doctors from the United States and Canada published a case study Nov. 9 in the

  • Cara Murez HealthDay Reporter
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  • November 10, 2022
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  • 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, breast and lung.

    According to a report in the journal Nature, the genes of these immune cells were edited...

    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 clinical trial at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania, in Phi...

    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 genetic disorder in which people have low levels of the

  • By Dennis Thompson HealthDay Reporter
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  • July 22, 2022
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  • 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, numbness or muscle weakness caused by damaged or malfunctioning nerves.

    Treatment of neuropathy can be challenging. For...

    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 epidermolysis bullosa (EB) are called butterfly children "because their skin...

    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 their own insulin, experts say.

    At least one patient - a 64-year-old Ohio man named

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  • February 25, 2022
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  • 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 people -- including Lyme disease, babesiosis and Rocky Mountain spotted fever -- but genetic knowledge about ticks is cu...

    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 skin conditions, progress in acne has been limited," said Catherine Smith, co-author of a

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  • February 8, 2022
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  • 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 has vanished, University of Pennsylvania doctors report.

    "I'm doing great right now. I'm still very active. I wa...

    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, according to clinical trial findings published Dec. 12 in the

  • Dennis Thompson HealthDay Reporter
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  • December 13, 2021
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  • 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 missing coagulation factor called FVIII.

    The current standard of care involves regular infusions of the FVIII protein...

    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 safe over two years. It also showed potential for beating the current standard treatment, stem cell transplantation.


    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 caused by the disease, which is a progressive disorder of the nervous system.

    But a Northwestern University team fo...

    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 system disorder formally known as adenosine deaminase--deficient severe combined immunodeficiency (ADA-SCID).


    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 receiving [anti-CD20] treatment to get a COVID-19 vaccine, which will prevent severe illness," said researcher E. John Wher...

    Pig Study Could Lead to Gene Therapy to Prevent Heart Failure

    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 heart attack damage.

    There is a long way to go before a similar gene therapy could be applied to human heart attac...

    Man Blind for 40 Years Regains Some Sight Through Gene Therapy

    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 retinitis pigmentosa, a genetic disorder that breaks down cells that absorb and convert light into brain signals.


    Gene Therapy Uses HIV to Rescue Kids Born Without Immune System

    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 dangerous, life-threatening infections from day-to-day activities as simple as going to school or playing with friends.


    Early Promise for Therapy Against Duchenne Muscular Dystrophy

    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 ability to regenerate after an injury.

    Muscle tissue is eventually replaced by fat and collagen. Many children wit...

    Could Gene Therapy Cure Sickle Cell Disease? Two New Studies Raise Hopes

    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 hemoglobin, said Dr. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America.

    Sickle cell dis...

    Could Gene Therapy Stem the Damage of Parkinson's?

    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 that the disease has already laid waste to significant portions of the brain by the time it is diagnosed," said researche...

    CRISPR Gene Editing Creates 'Designer' Immune Cells That Fight Cancer

    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-editing tool known as CRISPR into cancer treatment.

    CRISPR allows researchers to precisely "snip" bits of DNA wit...

    Researchers Alter Mosquitoes to Resist Dengue Infection

    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.

    This is the first time that mosquitoes have been genetically engineered to be resistant to all types of dengue, which c...

    Gene Therapy May Be Long-Term Cure for Type of Hemophilia

    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 years after their initial treatment, researchers reported Jan. 1 in the New England Journal of Medicine.


    'Designer Babies' a Long Way Off

    "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.

    But while recent live births resulting from embryonic CRISPR gene editing have re-focused attention on the issue, the...

    How Chinese Gene-Editing Could Backfire: Babies Might Have Shorter Lives

    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 the scientist used -- affecting a gene called CCR5 -- is associated with a shorter life span: People who carry two copies ...

    Gene Therapy May Help Fight Tough-to-Treat Blood Cancer

    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 there are treatments that can help people live with the disease for years.

    However, most people eventually pr...

    Gene Therapy Might Prove a Cure for 'Bubble Boy' Disease

    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 be a cure for X-linked severe combined immune deficiency (SCID-X1), widely known as "bubble boy disease."

    Ten ...