logo
Healthy babies born in Britain after scientists used DNA from three people to avoid genetic disease

Healthy babies born in Britain after scientists used DNA from three people to avoid genetic disease

Yahoo17-07-2025
LONDON (AP) — Eight healthy babies were born in Britain with the help of an experimental technique that uses DNA from three people to help mothers avoid passing devastating rare diseases to their children, researchers reported Wednesday.
Most DNA is found in the nucleus of our cells, and it's that genetic material — some inherited from mom, some from dad — that makes us who we are. But there's also some DNA outside of the cell's nucleus, in structures called mitochondria. Dangerous mutations there can cause a range of diseases in children that can lead to muscle weakness, seizures, developmental delays, major organ failure and death.
Testing during the in vitro fertilization process can usually identify whether these mutations are present. But in rare cases, it's not clear.
Researchers have been developing a technique that tries to avoid the problem by using the healthy mitochondria from a donor egg. They reported in 2023 that the first babies had been born using this method, where scientists take genetic material from the mother's egg or embryo, which is then transferred into a donor egg or embryo that has healthy mitochondria but the rest of its key DNA removed.
The latest research 'marks an important milestone,' said Dr. Zev Williams, who directs the Columbia University Fertility Center and was not involved in the work. 'Expanding the range of reproductive options … will empower more couples to pursue safe and healthy pregnancies.'
Using this method means the embryo has DNA from three people — from the mother's egg, the father's sperm and the donor's mitochondria — and it required a 2016 U.K. law change to approve it. It is also allowed in Australia but not in many other countries, including the U.S.
Experts at Britain's Newcastle University and Monash University in Australia reported in the New England Journal of Medicine Wednesday that they performed the new technique in fertilized embryos from 22 patients, which resulted in eight babies that appear to be free of mitochondrial diseases. One woman is still pregnant.
One of the eight babies born had slightly higher than expected levels of abnormal mitochondria, said Robin Lovell-Badge, a stem cell and developmental genetics scientist at the Francis Crick Institute who was not involved in the research. He said it was still not considered a high enough level to cause disease, but should be monitored as the baby develops.
Dr. Andy Greenfield, a reproductive health expert at the University of Oxford, called the work 'a triumph of scientific innovation,' and said the method of exchanging mitochondria would only be used for a small number of women for whom other ways of avoiding passing on genetic diseases, like testing embryos at an early stage, was not effective.
Lovell-Badge said the amount of DNA from the donor is insignificant, noting that any resulting child would have no traits from the woman who donated the healthy mitochondria. The genetic material from the donated egg makes up less than 1% of the baby born after this technique.
'If you had a bone marrow transplant from a donor … you will have much more DNA from another person,' he said.
In the U.K., every couple seeking a baby born through donated mitochondria must be approved by the country's fertility regulator. As of this month, 35 patients have been authorized to undergo the technique.
Critics have previously raised concerns, warning that it's impossible to know the impact these sorts of novel techniques might have on future generations.
'Currently, pronuclear transfer is not permitted for clinical use in the U.S., largely due to regulatory restrictions on techniques that result in heritable changes to the embryo," Williams, of Columbia, said in an email. 'Whether that will change remains uncertain and will depend on evolving scientific, ethical, and policy discussions."
For about a decade, Congress has included provisions in annual funding bills banning the Food and Drug Administration from accepting applications for clinical research involving techniques, 'in which a human embryo is intentionally created or modified to include a heritable genetic modification.'
But in countries where the technique is allowed, advocates say it could provide a promising alternative for some families.
Liz Curtis, whose daughter Lily died of a mitochondrial disease in 2006, now works with other families affected by them. She said it was devastating to be told there was no treatment for her eight-month-old baby and that death was inevitable.
She said the diagnosis 'turned our world upside down, and yet nobody could tell us very much about it, what it was or how it was going to affect Lily.' Curtis later founded the Lily Foundation in her daughter's name to raise awareness and support research into the disease, including the latest work done at Newcastle University.
'It's super exciting for families that don't have much hope in their lives,' Curtis said.
___
Ungar reported from Erie, Pennsylvania.
——-
The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
Maria Cheng And Laura Ungar, The Associated Press
Orange background

Try Our AI Features

Explore what Daily8 AI can do for you:

Comments

No comments yet...

Related Articles

EMA Recommends Eco-Friendly Inhaler for COPD
EMA Recommends Eco-Friendly Inhaler for COPD

Medscape

time19 minutes ago

  • Medscape

EMA Recommends Eco-Friendly Inhaler for COPD

The European Medicines Agency (EMA) has recommended a change in composition for Trixeo Aerosphere, also known as Riltrava Aerosphere. Both drugs are used for maintenance treatment in a subset of adults with moderate-to-severe chronic obstructive pulmonary disease (COPD). The recomposition involves replacing the existing gas propellant, HFA-134a, with a low global warming-potential (GWP) gas alternative called HFO-1234ze(E). The propellant is used for drug delivery and is not an active medicine. AstraZeneca claims that the new propellant will lead to a 99.9% reduction in GWP and an 85% reduction in greenhouse gas emissions compared with current propellants. The company adds that its carbon footprint is similar to that of dry powder inhalers. The reformulation was approved by British regulators in May and is currently under review in other countries such as China. COPD is an umbrella term for a group of lung conditions, including emphysema and chronic bronchitis. Symptoms include shortness of breath, frequent chest infections, and persistent wheezing. Estimates suggest that around 36.5 million people had COPD in Europe in 2020, and that this number will increase to 49.5 million by 2050. Trixeo Aerosphere and Riltrava Aerosphere have three active ingredients: budesonide, glycopyrronium, and formoterol fumarate dihydrate. Budesonide is a glucocorticosteroid that exerts rapid, dose-dependent, anti-inflammatory action on the airways when inhaled. Meanwhile, glycopyrronium is a long-acting muscarinic antagonist and formoterol fumarate dihydrate is a selective beta2-adrenergic agonist. Both result in bronchodilation. The reformulation comes as the EU phases out high GWP gases for environmental reasons. The EMA reported that studies confirm the safety and efficacy of the reformulated medicine are equivalent to those of the currently approved product. The associated studies will be made available in the variation assessment report following the European Commission's final decision, which is expected in the coming weeks. Annie Lennon is a medical journalist. Her writing appears on Medscape, WebMD, and Medical News Today, among other outlets.

NHS urges public to do one thing as doctors' strikes begin
NHS urges public to do one thing as doctors' strikes begin

Yahoo

timean hour ago

  • Yahoo

NHS urges public to do one thing as doctors' strikes begin

The NHS in the north west is urging people to do one thing as doctors' strikes begin. The region's NHS is asking people to come forward for care and 'use services wisely during industrial action' by resident doctors starting today (July 25). The five-day walk out by resident doctors, from 7am today, Friday, to 7am Wednesday, July 30, marks the twelfth strike from resident doctors since March 2023. READ MORE: 'We were relaxing by the pool at a luxury resort in Mexico when our holiday turned into a nightmare' READ MORE: Jay Slater inquest finally reveals exactly what happened to tragic teenager in Tenerife as cause of death confirmed It will result in 49 days of disruption to NHS services. Never miss a story with the MEN's daily Catch Up newsletter - get it in your inbox by signing up here During these strikes, all other NHS staff, including consultants and other specialist doctors, will still be working, and the focus of the NHS will be on ensuring as many services as possible continue to operate safely. Dr Michael Gregory, Regional Medical Director for NHS England in the North West, said: 'This period of industrial action will create significant challenges for the NHS and it is vital the public know how they can access the care they need during that time. 'It's important that people use NHS 111 online ( as their first port of call for all non-urgent health needs, as well as use their local GP practice and community pharmacies as usual. 'Urgent and emergency care services remain open during the industrial action period and people should call 999 or attend the accident and emergency department in the event of a life or limb threatening emergency.' 'We know from previous strikes that there will be significant disruption to patient appointments as a result of the strikes', said the region's NHS. There may be fewer doctors working during this time and they will need to prioritise life saving care. 'The NHS will do its best to only cancel appointments where it is necessary', health bosses have promised. Join our Family WhatsApp group HERE Dr Michael Gregory continued: 'I would urge people to please attend your appointment as planned. The NHS will contact you if your appointment needs to be rescheduled due to strike action. 'I'd like to thank the public for their continued support and our NHS staff who are going above and beyond to maintain safe patient services during this challenging period.' The NHS is also asking the public to play their part during industrial action by looking after themselves, loved ones and checking in on vulnerable family members and neighbours. For more information about industrial action and how you can use NHS services wisely during this time, visit: NHS England » Information for the public on industrial action.

Sarepta Therapeutics Acknowledges CHMP Negative Opinion for ELEVIDYS in the European Union
Sarepta Therapeutics Acknowledges CHMP Negative Opinion for ELEVIDYS in the European Union

Yahoo

timean hour ago

  • Yahoo

Sarepta Therapeutics Acknowledges CHMP Negative Opinion for ELEVIDYS in the European Union

Partner Roche will continue its dialogue with the European Medicines Agency to explore a potential path forward to make ELEVIDYS available to individuals living with Duchenne muscular dystrophy in the EU ELEVIDYS is the first and only disease-modifying gene therapy for Duchenne CAMBRIDGE, Mass., July 25, 2025--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, acknowledges that the Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the conditional marketing authorization (CMA) for ELEVIDYS (delandistrogene moxeparvovec) in ambulatory individuals ages three to seven years for the treatment of Duchenne muscular dystrophy (DMD). "While we are disappointed by the CHMP's negative opinion, we understand the urgent need for continued dialogue and collaboration to bring transformative therapies to people with Duchenne who live with a relentless disease that steals their mobility, independence and ultimately life – often by early adulthood," said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta. "Following the initial FDA approval of ELEVIDYS on June 22, 2023, the therapy has subsequently received regulatory approval in several other countries. In the U.S., we are actively working with the FDA to address recent safety questions. We remain committed to working with regulators to address outstanding questions on safety so that people living with Duchenne have access to this important therapy." ELEVIDYS is the first and only approved gene therapy targeting the underlying cause of disease that has consistently demonstrated stabilization or slowing of DMD disease progression, with durable effects on functional and biological outcomes and muscle health. While the primary endpoint was not met in EMBARK after one year, ELEVIDYS showed clinically meaningful and statistically significant improvements across important secondary endpoints of functional outcome measures when compared to placebo. Longer term efficacy data were also submitted to EMA, including two-year results from the EMBARK study and three-year pooled efficacy analysis from three other ELEVIDYS studies that showed clinically meaningful improvements across key measures of motor function. One-year data from part one of the EMBARK study were published in Nature Medicine in October 2024, and results from year two were shared at this year's Muscular Dystrophy Association Clinical & Scientific Conference in Dallas. Quantitative muscle MR (magnetic resonance) outcomes from part 1 of EMBARK were published in JAMA Neurology in May 2025. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world. Regulatory approval and commercialization of ELEVIDYS in Japan is through Chugai Pharmaceuticals via its alliance with Roche. About ELEVIDYS (delandistrogene moxeparvovec-rokl)ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated in U.S. for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval in the U.S. based on expression of ELEVIDYS micro-dystrophin (noted hereafter as "micro-dystrophin") in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). U.S. IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient's clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Preexisting Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia. Report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information. About Sarepta TherapeuticsSarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website at We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking StatementsThis statement contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "will," "may," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, discussions with regulators and the prospects for approvals or continued approvals, as applicable, of ELEVIDYS and the potential benefits and risks of ELEVIDYS. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences; the possible impact of regulatory decisions by, and any halts imposed by, regulatory agencies on our business; and those risks identified under the heading "Risk Factors" in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law. Source: Sarepta Therapeutics, Inc. View source version on Contacts Investor Contact: Ian Estepan617-274-4052iestepan@ Media Contacts: Tracy Sorrentino617-301-8566tsorrentino@ Kara Hoeger617-710-3898KHoeger@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data

DOWNLOAD THE APP

Get Started Now: Download the App

Ready to dive into a world of global content with local flavor? Download Daily8 app today from your preferred app store and start exploring.
app-storeplay-store