Friday, 31 Oct 2025
  • Contact
  • Privacy Policy
  • Terms & Conditions
  • DMCA
logo logo
  • World
  • Politics
  • Crime
  • Economy
  • Tech & Science
  • Sports
  • Entertainment
  • More
    • Education
    • Celebrities
    • Culture and Arts
    • Environment
    • Health and Wellness
    • Lifestyle
  • 🔥
  • Trump
  • VIDEO
  • House
  • White
  • ScienceAlert
  • Trumps
  • Watch
  • man
  • Health
  • Season
Font ResizerAa
American FocusAmerican Focus
Search
  • World
  • Politics
  • Crime
  • Economy
  • Tech & Science
  • Sports
  • Entertainment
  • More
    • Education
    • Celebrities
    • Culture and Arts
    • Environment
    • Health and Wellness
    • Lifestyle
Follow US
© 2024 americanfocus.online – All Rights Reserved.
American Focus > Blog > Tech and Science > Baby with rare disease given world-first personal CRISPR gene therapy
Tech and Science

Baby with rare disease given world-first personal CRISPR gene therapy

Last updated: May 15, 2025 5:00 pm
Share
Baby with rare disease given world-first personal CRISPR gene therapy
SHARE

A Glimpse into the Future of Medicine: Baby Receives Bespoke CRISPR Gene-Editing Treatment

Baby KJ after a gene-editing infusion with researchers Rebecca Ahrens-Nicklas and Kiran Musunuru

Baby KJ after a gene-editing infusion with researchers Rebecca Ahrens-Nicklas and Kiran Musunuru

Children’s Hospital of Philadelphia

A groundbreaking moment in medical history has occurred as a baby boy with a life-threatening genetic condition became the first person to undergo a personalized CRISPR gene-editing treatment. This innovative approach provides a glimpse into the potential future of medicine.

Lead researcher, Rebecca Ahrens-Nicklas from the Children’s Hospital of Philadelphia, Pennsylvania, revealed that this treatment marks the first instance of a gene-editing therapy tailored to correct a disease-causing mutation unique to an individual. While the baby, known as KJ, is showing promising early signs of improvement, the full extent of the treatment’s effectiveness is still being evaluated.

The research team wasted no time in sharing their findings in the hopes of inspiring others in the medical field. Kiran Musunuru from the University of Pennsylvania expressed optimism about the future implications of this milestone. He emphasized, “I don’t think I’m exaggerating when I say that this is the future of medicine. This is the first step towards the use of gene-editing therapies to treat a wide variety of rare genetic disorders.”

The baby, KJ, inherited mutations in both copies of a gene responsible for producing a crucial liver enzyme called CPS1. The absence of this enzyme leads to the accumulation of ammonia in the bloodstream, causing brain damage when proteins are broken down. Ahrens-Nicklas highlighted the severity of CPS1 deficiency, stating that more than half of affected children do not survive.

Developing treatments that specifically target the liver, Ahrens-Nicklas and Musunuru swiftly devised a base-editing therapy using CRISPR technology to correct one of KJ’s faulty CPS1 genes. The urgency of KJ’s condition prompted the researchers to seek expedited approval from US regulators, resulting in swift authorization from the FDA.

See also  Trump Administration Axes Dozens at CDC — Entire Washington Office, “Disease Detectives,” and Senior Scientists Among Those Caught in Massive Federal Layoffs | The Gateway Pundit | by Jim Há´Źft

KJ received multiple doses of the gene-editing treatment, with noticeable improvements in his ability to tolerate protein intake and reduced reliance on other medications to manage his condition. While the ideal scenario involves treating children at an even earlier stage to prevent long-term complications, the potential of gene-editing therapies to revolutionize the management of rare genetic disorders is evident.

Unlike traditional gene-editing treatments designed for broader applications, personalized therapies like KJ’s are expected to incur higher costs initially. However, Musunuru anticipates a significant reduction in expenses as advancements in technology and production efficiency evolve. The shift towards a platform-based approach by regulators, granting approval for therapies targeting various mutations within the same gene, signifies a pivotal change in the landscape of personalized medicine.

Embracing genome editing with CRISPR as a scalable solution for treating rare diseases, Nick Meade from Genetic Alliance UK emphasized the transformative impact of such approaches. This groundbreaking achievement not only offers hope to families affected by rare genetic conditions but also paves the way for a more accessible and effective treatment paradigm.

KJ with his family after the treatment

KJ with his family after the treatment

Children’s Hospital of Philadelphia

The Benefits of Meditation for Mental Health

Meditation has been practiced for thousands of years by people all over the world, and in recent years, it has gained mainstream popularity as a tool for improving mental health. Research has shown that regular meditation practice can have a number of benefits for mental health, including reducing stress, anxiety, and depression, improving focus and concentration, and increasing feelings of well-being and happiness.

See also  This 3-Step Hack Will Help Students Make Cheap, Healthy Meals

One of the key benefits of meditation is its ability to reduce stress. When we are stressed, our bodies go into fight or flight mode, releasing hormones like cortisol and adrenaline that can be harmful if they are constantly being produced. Meditation has been shown to reduce the production of these stress hormones, leading to a decrease in stress levels and an overall sense of calm and relaxation.

In addition to reducing stress, meditation can also help to alleviate symptoms of anxiety and depression. Studies have shown that meditation can lower levels of anxiety and depression by changing the way our brains respond to stress and negative emotions. By practicing mindfulness and focusing on the present moment, individuals can learn to let go of negative thoughts and feelings, leading to a greater sense of well-being and emotional balance.

Meditation has also been shown to improve focus and concentration, which can be particularly beneficial for those who struggle with attention deficit disorders or other cognitive impairments. By training the mind to focus on one thing at a time, meditation can help to increase mental clarity and improve cognitive function.

Furthermore, regular meditation practice can increase feelings of happiness and well-being. By cultivating a sense of gratitude, compassion, and self-awareness, individuals can learn to appreciate the present moment and find joy in simple pleasures. This can lead to an overall increase in life satisfaction and a greater sense of fulfillment.

Overall, the benefits of meditation for mental health are numerous and well-documented. By incorporating a regular meditation practice into your daily routine, you can experience reduced stress, anxiety, and depression, improved focus and concentration, and increased feelings of happiness and well-being. Whether you are new to meditation or have been practicing for years, there is no doubt that it can have a positive impact on your mental health and overall quality of life.

See also  Universal Audio Standard Series microphones review: These may be the only drum mics you’ll ever need
TAGGED:babyCRISPRdiseasegenePersonalRareTherapyWorldFirst
Share This Article
Twitter Email Copy Link Print
Previous Article Critic Renounces NYU Degrees After School Punishes Pro-Palestine Student  Critic Renounces NYU Degrees After School Punishes Pro-Palestine Student 
Next Article Walmart can absorb tariffs, fmr. U.S. CEO Simon questions price hikes Walmart can absorb tariffs, fmr. U.S. CEO Simon questions price hikes
Leave a comment

Leave a Reply Cancel reply

Your email address will not be published. Required fields are marked *

Popular Posts

Idaho Bans “Everyone Is Welcome Here” Posters. Teachers Want to Know—Who Isn’t?

This summer, Idaho made headlines as it joined a growing number of states implementing restrictions…

July 15, 2025

The Queer History of Central Park’s Bethesda Fountain

The article is part of Hyperallergic’s 2025 Pride Month series, shedding light on key moments…

June 18, 2025

Bollywood actress Wamiqa Gabbi chooses between Virat Kohli’s cover drive and Rohit Sharma’s pull shot [Watch]

Bollywood actress Wamiqa Gabbi recently made an interesting choice between Rohit Sharma's pull shot and…

April 29, 2025

WWE champion beats second-generation star; confronts ‘Baby’ Randy Orton ahead of major title match

A surprising turn of events unfolded on WWE NXT as Myles Borne, the current champion,…

May 13, 2025

Banana Company Artwashes Its Bloody Legacy at Art Basel Miami Beach

Chiquita's recent promotion on Instagram offering free tickets to Art Basel Miami Beach has sparked…

December 9, 2024

You Might Also Like

Trump’s Baffling Call for Resuming U.S. Nuclear Tests
Tech and Science

Trump’s Baffling Call for Resuming U.S. Nuclear Tests

October 31, 2025
Nanotyrannus: Dinosaur skeleton settles long debate over ‘tiny T. rex’ fossils
Tech and Science

Nanotyrannus: Dinosaur skeleton settles long debate over ‘tiny T. rex’ fossils

October 30, 2025
A Signal Hidden Deep in Your Brain Could Track Parkinson’s Symptoms : ScienceAlert
Tech and Science

A Signal Hidden Deep in Your Brain Could Track Parkinson’s Symptoms : ScienceAlert

October 30, 2025
Scenes from JS Disrupt | JS
Tech and Science

Scenes from JS Disrupt | JS

October 30, 2025
logo logo
Facebook Twitter Youtube

About US


Explore global affairs, political insights, and linguistic origins. Stay informed with our comprehensive coverage of world news, politics, and Lifestyle.

Top Categories
  • Crime
  • Environment
  • Sports
  • Tech and Science
Usefull Links
  • Contact
  • Privacy Policy
  • Terms & Conditions
  • DMCA

© 2024 americanfocus.online –  All Rights Reserved.

Welcome Back!

Sign in to your account

Lost your password?