Articles for Gene Therapy

Our son's terrifying illness is so rare only 28 other children have it... now we're on a quest to find a cure
1 week, 6 days ago

Our son's terrifying illness is so rare only 28 other children have it... now we're on a quest to find a cure

Daily Mail  

The photographs of Angus Powell when he was just six months old are joyful. Now three, Angus's future remains far from certain, but Sophie and Oli were told his symptoms place him at the milder end of the scale, which has given them hope. Angus was around six months old when he suffered the first of what would become a …

Children Hope Child Families Gene Therapy Lola Months
GST council approves key reforms, defers decision on insurance premium tax cuts
3 weeks ago

GST council approves key reforms, defers decision on insurance premium tax cuts

India TV News  

The Goods and Services Tax Council, chaired by Finance Minister Nirmala Sitharaman, made significant decisions in its 55th meeting, aimed at streamlining the taxation system and curbing tax evasion. Other exemptions and clarifications Third-Party Motor Vehicle Premiums: Contributions made by general insurance companies to the Motor Vehicle Accident Fund, established under the Motor Vehicles Act, 1988, have been exempted from …

Tax Services Council Gene Therapy Fund Gst Goods
India’s first gene therapy offers new hope for haemophilia patients
3 weeks, 4 days ago

India’s first gene therapy offers new hope for haemophilia patients

New Indian Express  

Results of the study conducted on the patients have been published in the recent edition of New England Journal of Medicine. The results of this clinical study reveal a new opportunity for gene therapy that can be offered to all haemophilia-A patients, possibly at an early age,” Dr Srivastava said. Physicians believe that the new gene therapy developed by CMC …

Therapy Gene Patients Gene Therapy Factor Haemophilia Factor Viii
How can gene therapy cure haemophilia A? | Explained
3 weeks, 6 days ago

How can gene therapy cure haemophilia A? | Explained

The Hindu  

The story so far: Medical researchers at the Christian Medical College, Vellore have reported successfully applying gene-therapy to treat severe haemophilia A, a rare, hereditary condition that results from a faulty gene which triggers severe, spontaneous and fatal bleeding episodes. As the disease is a condition resulting from low levels of clotting factor — necessary to prevent bleeding — replacement …

India Treatment Therapy Gene Gene Therapy Bleeding Vellore
India makes big breakthrough human gene therapy in treating genetic disorder Hemophilia A
1 month ago

India makes big breakthrough human gene therapy in treating genetic disorder Hemophilia A

India Today  

India has achieved a major breakthrough in treating severe Hemophilia A through gene therapy. In a groundbreaking study conducted by the Centre for Stem Cell Research at Christian Medical College, Vellore, five patients who received the therapy experienced zero bleeding episodes over an extended period. The therapy enabled the participants to produce Factor VIII, a protein critical for blood clotting, …

India Therapy Gene Patients Gene Therapy Factor Viii
CGT opening-up to spur China's bio-medical industry
1 month, 2 weeks ago

CGT opening-up to spur China's bio-medical industry

China Daily  

Employees perform sample pre-processing for use in sequencing analysis in Suzhou, Jiangsu province, on Nov 12. The measures were detailed in a guideline to support Suzhou Industrial Park to become a pilot area for opening-up and innovation throughout the biopharmaceutical industry chain, encouraging qualified institutions in the Suzhou section of the China Pilot Free Trade Zone to conduct clinical research …

China Industry Therapy Gene Therapy Shanghai Jiangsu Zhang
CGT opening-up to spur China's bio-medical industry
1 month, 2 weeks ago

CGT opening-up to spur China's bio-medical industry

China Daily  

Employees perform sample pre-processing for use in sequencing analysis in Suzhou, Jiangsu province, on Nov 12. The measures were detailed in a guideline to support Suzhou Industrial Park to become a pilot area for opening-up and innovation throughout the biopharmaceutical industry chain, encouraging qualified institutions in the Suzhou section of the China Pilot Free Trade Zone to conduct clinical research …

China Industry Therapy Gene Therapy Shanghai Jiangsu Zhang
RNA editing is the next frontier in gene therapy – here’s what you need to know
1 month, 2 weeks ago

RNA editing is the next frontier in gene therapy – here’s what you need to know

Raw Story  

The United States Food and Drug Administration has just approved the first-ever clinical trial that uses CRISPR-Cas13 RNA editing. Thanks to recent advances, we now have the tools to directly change someone’s DNA – this has paved the way for gene editing as a type of gene therapy. RNA editing doesn’t permanently change a person’s DNA, but rather alters the …

Trial Blood Gene Therapy Editing Dna Eye Amd
Haemophilia care: Here's how benefits and risks related to treatment of haemophilia can be balanced
5 months, 1 week ago

Haemophilia care: Here's how benefits and risks related to treatment of haemophilia can be balanced

Hindustan Times  

Haemophilia is a hereditary disorder that affects the ability of the body to form blood clots because of deficiency of factor VIII or IX but people with haemophilia can have active, meaningful lives provided they receive a regimen of factor replacement along with a well-organised exercise regimen to strengthen the muscle groups around major joints. It's a collaborative effort between …

Treatment Therapy Patients Gene Therapy Treatments Factors Risks
Takeaways from AP’s report on access to gene therapies for rare diseases
6 months, 3 weeks ago

Takeaways from AP’s report on access to gene therapies for rare diseases

Associated Press  

The promise of gene therapy looms large for families dealing with rare, genetic disorders. His mother Robin Alderman recalls one doctor saying: “Basically, your son’s only chance at a cure is going to be gene therapy.” He also told her researchers weren’t then accepting U.S. residents into a clinical trial for the treatment, which “just kind of broke my heart,” …

Therapy Gene Rare Diseases Gene Therapy Million Henderson Therapies
Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate
6 months, 3 weeks ago

Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate

Associated Press  

Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. “I just feel responsible to help them.” Families’ pain Camden Alderman, 21, who has a rare disease called Wiskott-Aldrich syndrome, holds some of the drugs and medical equipment he uses near his home in Greensboro, N.C., Wednesday, June 12, 2024. While he doesn’t …

Europe Disease Rare Therapy Gene Patients Families
Bryan Johnson ‘editing’ his DNA at remote island to live forever. Cost: $20,000
6 months, 3 weeks ago

Bryan Johnson ‘editing’ his DNA at remote island to live forever. Cost: $20,000

Hindustan Times  

Bryan Johnson said that he has ventured to the secluded island of Roatan off the coast of Honduras for gene therapy which he thinks can help reverse his age. In a YouTube video titled “I Edited My DNA On A Secret Island ”, Bryan Johnson said, "I’m traveling to a remote island for an extreme medical procedure that could change …

Therapy Gene Gene Therapy Johnson Bryan Bryan Johnson Live Forever
Health Matters newsletter: Hark! Good news on the HIV vaccine front
6 months, 3 weeks ago

Health Matters newsletter: Hark! Good news on the HIV vaccine front

The Hindu  

We could start with the NEET mess again, and there is indeed much to report since our last health newsletter. A collaborative survey 1,482 TB patients across four States conducted by the George Institute for Global Health, New Delhi, along with the Indira Gandhi Government Medical College, and the London School of Hygiene and Tropical Medicine, U.K., highlighted the severe …

Health India Karnataka Women Test Patients Gene Therapy
Scientifically Speaking | A groundbreaking gene therapy trial enables deaf children to hear for the first time
7 months ago

Scientifically Speaking | A groundbreaking gene therapy trial enables deaf children to hear for the first time

Hindustan Times  

For most of us, the joys of hearing a loved one’s voice or the simple pleasure of listening to music is a given. This innovative treatment, reported on June 5 in the scientific journal Nature Medicine, delivered a working copy of a crucial gene to the inner ear to allow these deaf children to hear for the first time. One …

Children Treatment Loss Trial Therapy Gene Gene Therapy
Groundbreaking 'cure' for deafness could be on the way after two children born with impaired hearing listen to music for first time thanks to gene therapy
7 months ago

Groundbreaking 'cure' for deafness could be on the way after two children born with impaired hearing listen to music for first time thanks to gene therapy

Daily Mail  

Two children who were born deaf can now listen and dance to music after undergoing ground-breaking gene therapy. Dr Yilai Shu from Eye & ENT Hospital of Fudan University examining a young patient The DFNB9 strain of deafness is caused by a failure to produce a protein known as otoferlin But two showed more advanced improvements and were even able …

Therapy Gene Gene Therapy Hearing Ear Hear Both Ears
U.K. girl's hearing restored after groundbreaking gene therapy
8 months ago

U.K. girl's hearing restored after groundbreaking gene therapy

The Hindu  

An 18-month old British girl who was born completely deaf is believed to be the youngest person to have their hearing restored after undergoing groundbreaking new gene therapy. But U.K. ear surgeon Manohar Bance said the toddler, Opal, was the first person in the world to receive therapy developed by U.S. biotech firm Regeneron and "the youngest globally that's been …

Therapy Gene Gene Therapy Cambridge Opal Bance Hearing Restored
Gene therapy trials treat inherited blindness and deafness
8 months ago

Gene therapy trials treat inherited blindness and deafness

Hindustan Times  

Scientists have restored vision and hearing in people born with congenital blindness or deafness in two separate gene therapy trials A new CRISPR gene editing called EDIT-101 has successfully restored vision in some patients with congenital blindness A British toddler born deaf has become the first person to have their hearing restored via gene therapy. CRISPR gene therapy restores vision …

Vision Therapy Gene Gene Therapy Treat Participants Crispr
UK toddler, born completely deaf, hears for first time after pioneering gene therapy: 'It was bonkers'
8 months ago

UK toddler, born completely deaf, hears for first time after pioneering gene therapy: 'It was bonkers'

Hindustan Times  

A UK toddler's hearing was restored after undergoing the world's first-ever pioneering gene therapy trial. According to reports by The Guardian, the toddler named Opal Sandy, from Oxfordshire, was born completely deaf due to auditory neuropathy, a rare genetic condition that disrupts nerve impulses travelling from the inner ear to the brain due to a gene defect. The CHORD Gene …

Children Trial Therapy Gene Gene Therapy Deaf Ear
Deaf girl is cured in world first gene therapy trial
8 months ago

Deaf girl is cured in world first gene therapy trial

The Independent  

Sign up for our free Health Check email to receive exclusive analysis on the week in health Get our free Health Check email Please enter a valid email address Please enter a valid email address SIGN UP I would like to be emailed about offers, events and updates from The Independent. Now, thanks to a “one and done” gene therapy …

Treatment Trial Normal Gene Therapy Hearing James Deaf
Pfizer reports patient death in Duchenne gene therapy study
8 months ago

Pfizer reports patient death in Duchenne gene therapy study

The Hindu  

A young patient died due to cardiac arrest after receiving Pfizer's experimental gene therapy being tested in a mid-stage trial for a muscle-wasting disorder called Duchenne muscular dystrophy, the drugmaker told Reuters on May 7. "A fatal serious adverse event was reported as cardiac arrest for a participant in the Phase 2 DAYLIGHT study," a company spokesperson told Reuters in …

Therapy Gene Gene Therapy Reuters Pfizer Dmd Duchenne
German firm Miltenyi Biotec opens office, plans Cell and Gene Therapy CoE
10 months, 2 weeks ago

German firm Miltenyi Biotec opens office, plans Cell and Gene Therapy CoE

The Hindu  

German firm Miltenyi Biotec on Tuesday announced the launch of operations in India with the opening of an office in Hyderabad and further plans to invest in a first-of-its-kind Cell and Gene Therapy centre of excellence for Indian academia and industry. Scientists, researchers, industry experts and clinicians can get classroom to hands-on training on cell and gene therapy approaches, from …

India Car Therapy Gene Therapy Cell Coe Cgt
Gene therapy improves advanced heart failure in animal model: Study
10 months, 3 weeks ago

Gene therapy improves advanced heart failure in animal model: Study

Hindustan Times  

During a heart attack, blood stops flowing into the heart. Gene therapy improves advanced heart failure in animal model: Study The heart muscle does not regenerate, instead, it replaces dead tissue with a scar made of cells called fibroblasts that do not help the heart pump. "In this study published in the journal Cardiovascular Research, we show that advanced heart …

Failure Gene Heart Gene Therapy Heart Failure Improve Mohamed
Breakthrough gene therapy treatment restores hearing in children born deaf
11 months, 2 weeks ago

Breakthrough gene therapy treatment restores hearing in children born deaf

The Independent  

Sign up for our free Health Check email to receive exclusive analysis on the week in health Get our free Health Check email Get our free Health Check email SIGN UP I would like to be emailed about offers, events and updates from The Independent. Read our privacy policy A breakthrough gene therapy treatment in China has restored hearing in …

Children Treatment Loss Gene Gene Therapy Hearing Deaf
Experimental gene therapy allows kids with inherited deafness to hear
11 months, 2 weeks ago

Experimental gene therapy allows kids with inherited deafness to hear

Associated Press  

Gene therapy has allowed several children born with inherited deafness to hear. “No treatment could reverse hearing loss … That’s why we were always trying to develop a therapy,” said Zheng-Yi Chen of Boston’s Mass Eye and Ear, a senior author of the study published Wednesday in the journal Lancet. ” Columbia University’s Dr. Lawrence Lustig, who is involved in …

Children Kids Therapy Gene Gene Therapy Philadelphia Chen