Internewscast Journal
  • Home
  • US News
  • Local News
  • Health
  • People
  • Guest Post
  • Support Our Cause
Internewscast Journal
  • Home
  • US News
  • Local News
  • Health
  • People
  • Guest Post
  • Support Our Cause
Home Local news Gene Editing Transforms Health for Critically Ill Infant; Experts Hopeful for Future Widespread Applications
  • Local news

Gene Editing Transforms Health for Critically Ill Infant; Experts Hopeful for Future Widespread Applications

    Gene editing helped a desperately ill baby thrive. Scientists say it could someday treat millions
    Up next
    Kristi Noem wants migrants to battle for citizenship on reality TV
    Kristi Noem Proposes Reality TV Competition for Migrants to Earn Citizenship
    Published on 15 May 2025
    Author
    Internewscast
    Tags
    • baby,
    • Carlos Moraes,
    • Could,
    • desperately,
    • editing,
    • gene,
    • Gene Editing,
    • health,
    • helped,
    • ill,
    • Jude Children,
    • millions,
    • Nicole Muldoon,
    • Rebecca Ahrens-Nicklas,
    • say,
    • science,
    • scientists,
    • someday,
    • thrive,
    • treat,
    • U.S. news
    Share this @internewscast.com
    FacebookXRedditPinterest

    A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him.

    In a newly published study, researchers have highlighted the case of a patient who is among the first to receive successful treatment through a personalized therapy. This approach aims to correct a minor yet crucial genetic error that proves fatal for half of the infants affected. Although it might take some time for such tailored treatments to be widely accessible, medical professionals are optimistic that one day this technology could benefit the millions with rare conditions often overlooked despite advances in genetic medicine.

    “This marks an initial step towards utilizing gene editing therapies to address a broad range of rare genetic disorders that currently lack definitive medical treatments,” stated Dr. Kiran Musunuru, a gene editing specialist from the University of Pennsylvania and co-author of the study, which was released on Thursday in the New England Journal of Medicine.

    KJ Muldoon from Clifton Heights, Pennsylvania, is the infant in focus, and he is one among 350 million people globally suffering from rare diseases, the majority of which are genetic. Shortly after his birth, he was found to have severe CPS1 deficiency, a condition some experts believe affects roughly one in a million newborns. These infants are deficient in an enzyme necessary for removing ammonia from their bodies, leading to its accumulation in the blood, which can become toxic. A liver transplant could be a potential solution for some of these cases.

    Knowing KJ’s odds, parents Kyle and Nicole Muldoon, both 34, worried they could lose him.

    “We were, like, you know, weighing all the options, asking all the questions for either the liver transplant, which is invasive, or something that’s never been done before,” Nicole said.

    “We prayed, we talked to people, we gathered information, and we eventually decided that this was the way we were going to go,” her husband added.

    Within six months, the team at Children’s Hospital of Philadelphia and Penn Medicine, along with their partners, created a therapy designed to correct KJ’s faulty gene. They used CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020. Instead of cutting the DNA strand like the first CRISPR approaches, doctors employed a technique that flips the mutated DNA “letter” — also known as a base — to the correct type. Known as “base editing,” it reduces the risk of unintended genetic changes.

    It’s “very exciting” that the team created the therapy so quickly, said gene therapy researcher Senthil Bhoopalan at St. Jude Children’s Research Hospital in Memphis, who wasn’t involved in the study. “This really sets the pace and the benchmark for such approaches.”

    In February, KJ got his first IV infusion with the gene editing therapy, delivered through tiny fatty droplets called lipid nanoparticles that are taken up by liver cells.

    While the room was abuzz with excitement that day, “he slept through the entire thing,” recalled study author Dr. Rebecca Ahrens-Nicklas, a gene therapy expert at CHOP.

    After follow-up doses in March and April, KJ has been able to eat more normally and has recovered well from illnesses like colds, which can strain the body and exacerbate symptoms of CPS1. The 9 ½-month old also takes less medication.

    Considering his poor prognosis earlier, “any time we see even the smallest milestone that he’s meeting – like a little wave or rolling over – that’s a big moment for us,” his mother said.

    Still, researchers caution that it’s only been a few months. They’ll need to watch him for years.

    “We’re still very much in the early stages of understanding what this medication may have done for KJ,” Ahrens-Nicklas said. “But every day, he’s showing us signs that he’s growing and thriving.”

    Researchers hope what they learn from KJ will help other rare disease patients.

    Gene therapies, which can be extremely expensive to develop, generally target more common disorders in part for simple financial reasons: more patients mean potentially more sales, which can help pay the development costs and generate more profit. The first CRISPR therapy approved by the U.S. Food and Drug Administration, for example, treats sickle cell disease, a painful blood disorder affecting millions worldwide.

    Musunuru said his team’s work — funded in part by the National Institutes of Health — showed that creating a custom treatment doesn’t have to be prohibitively expensive. The cost was “not far off” from the $800,000-plus for an average liver transplant and related care, he said.

    “As we get better and better at making these therapies and shorten the time frame even more, economies of scale will kick in and I would expect the costs to come down,” Musunuru said.

    Scientists also won’t have to redo all the initial work every time they create a customized therapy, Bhoopalan said, so this research “sets the stage” for treating other rare conditions.

    Carlos Moraes, a neurology professor at the University of Miami who wasn’t involved with the study, said research like this opens the door to more advances.

    “Once someone comes with a breakthrough like this, it will take no time” for other teams to apply the lessons and move forward, he said. “There are barriers, but I predict that they are going to be crossed in the next five to 10 years. Then the whole field will move as a block because we’re pretty much ready.”

    ———-

    The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Science and Educational Media Group and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.

    Copyright 2025 The Associated Press. All rights reserved. This material may not be published, broadcast, rewritten or redistributed without permission.

    Share this @internewscast.com
    FacebookXRedditPinterest
    You May Also Like
    Austin House closes, several families displaced
    • Local news

    Austin House Shutters, Displacing Several Families

    SAVANNAH, Ga. () – The Tom D. Austin house is closing permanently,…
    • Internewscast
    • July 1, 2025
    EPA says it will delay pollution rules for coal plants
    • Local news

    EPA to Postpone Enforcement of Pollution Regulations for Coal Plants

    The Trump administration says that it plans to delay and potentially loosen…
    • Internewscast
    • July 1, 2025
    Solar minigrid brings light and hope to a Goma neighborhood, offering blueprint for rest of Congo
    • Local news

    Solar Minigrid Illuminates Goma Neighborhood, Provides Model for Congo

    GOMA – Street lights erase the shadows where attackers once hid. Noisy,…
    • Internewscast
    • July 1, 2025
    Trump to visit 'Alligator Alcatraz' detention center this week
    • Local news

    Trump Scheduled to Visit Notorious ‘Alligator Alcatraz’ Detention Center This Week

    (NewsNation) — President Donald Trump is anticipated to pay a visit to…
    • Internewscast
    • June 30, 2025
    Senate to begin 'vote-a-rama' on GOP bill and two firefighters killed in ambush: Morning Rundown
    • Local news

    Senate Begins Marathon Voting Session on GOP Bill; Two Firefighters Tragically Killed in Ambush: Morning Update

    Today, the Senate will initiate its ‘vote-a-rama’ on Donald Trump’s policy bill.…
    • Internewscast
    • June 30, 2025
    Joint press conference with Richmond and Burke Sheriff's Offices on death of Xavion Wimberly
    • Local news

    Richmond and Burke Sheriff’s Offices Hold Joint Press Conference Regarding Xavion Wimberly’s Death

    AUGUSTA, Ga. () – Significant announcements and substantial financial developments emerged from…
    • Internewscast
    • July 1, 2025
    Texas man admits to killing missing American Airlines flight attendant: Docs
    • Local news

    Texas Resident Confesses to Murder of Missing American Airlines Flight Attendant: Court Documents Reveal

    TARRANT COUNTY, Texas (KFDX/KJTL) A Fort Worth man has confessed to killing…
    • Internewscast
    • July 1, 2025

    “Concert Chaos in Portland: Fans Face Long Waits in Lines”

    PORTLAND, Ore. (KOIN) Thousands of Post Malone and Jelly Roll fans were…
    • Internewscast
    • June 30, 2025
    Jury due to begin deliberating in Sean ‘Diddy’ Combs’ sex trafficking trial
    • Local news

    Jury Set to Start Deliberations in Sex Trafficking Case Involving Sean “Diddy” Combs

    NEW YORK (AP) — A jury will begin deliberations on Monday in…
    • Internewscast
    • June 30, 2025
    Storm Team 3: Hot with more storms ahead of the Fourth
    • Local news

    Storm Team 3: Heat Continues with More Storms Forecasted Before Fourth of July

    The week kicked off with hot and humid weather. Afternoon temperatures climbed…
    • Internewscast
    • July 1, 2025
    13 charged in 'largest healthcare fraud investigation in DOJ history'
    • Local news

    13 Individuals Charged in Major Healthcare Fraud Case, the Largest in DOJ’s History

    (WXIN/WTTV) 13 Indiana residents have been arrested and charged in what the…
    • Internewscast
    • June 30, 2025
    Death toll rises to 36 following an explosion and fire at a southern India pharmaceutical factory
    • Local news

    Tragic Blast and Fire at Southern India Pharma Factory Claims 36 Lives

    Copyright 2025 The Associated Press. All rights reserved. Rescue workers look for…
    • Internewscast
    • July 1, 2025
    How To Watch Wimbledon 2025
    • US

    Your Guide to Watching Wimbledon 2025

    Get ready, tennis enthusiasts: the 2025 Wimbledon Championships are now underway! With…
    • Internewscast
    • July 1, 2025
    Thailand's Prime Minister Paetongtarn Shinawatra arrives at Government House for a cabinet meeting in Bangkok, Thailand.
    • AU

    Thailand’s Prime Minister Removed After Controversial Phone Call Leak

    Thailand’s Constitutional Court has temporarily removed Prime Minister…
    • Internewscast
    • July 1, 2025
    Mugshot of Jocelyn Sanroman.
    • US

    26-Year-Old Teacher Faces Charges for Alleged Sexual Relationship with 16-Year-Old Student Following Confession to Colleague

    A TEACHER has been charged with having sex with a 16-year-old student…
    • Internewscast
    • July 1, 2025

    Israel to Investigate Strike on Gaza Beach Cafe Resulting in 24 Palestinian Deaths

    The Israeli army said Tuesday that it launched a review into a…
    • Internewscast
    • July 1, 2025
    Internewscast Journal
    • Home
    • Privacy Policy
    • DMCA Notice
    • Terms and Conditions
    • Guest Post
    • Support Our Cause
    Copyright 2023. All Right Reserverd.