• Adults Only Website 18+

    If you are under 18 you are not permitted to submit personal information to us or use this website. If discovered you will be banned.

    We will ban and report anyone posting illegal content.

    We will ban any forum user who breaks our terms.

    Freedom of speech should be wide open as long as it doesn't incite violence.

    We have a 15 year old thriving community here with 400,000+ members and hundreds of people online at any given moment, we encourage you to join!, there are 1000's of topics to discuss. Please be aware before registering and read our terms of service and privacy policy.

    By dismissing this notice and proceeding, you agree to the above.

Baby saved by gene-editing therapy takes 1st steps ahead of Christmas

From ABC News:

The actual baby:
IMG_3856.webp


The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead of Christmas.

KJ Muldoon is walking and getting ready to celebrate the holiday season at home with his parents and three siblings.

KJ was born last year with a genetic disorder called carbamoyl-phosphate synthetase 1 deficiency. Also known as CPS1 deficiency, the metabolic condition affects only 1 in 1.3 million babies and often leads to life-threatening outcomes.

——-

Not in article but the wiki on disease if you are interested:
Carbamoyl phosphate synthetase I deficiency - Wikipedia

———

When he was 6 months old, doctors began giving KJ a groundbreaking new treatment -- a personalized CRISPR gene editing therapy at Children's Hospital of Philadelphia.

The gene-editing therapy works like a "molecular spell-checker,” finding and fixing the specific genetic error causing his condition.

After three infusions and after spending the first 10 months of his life in a hospital, KJ was dischargedand sent home in June.



Since then, KJ has continued to grow and thrive with his family, celebrating his first birthday at home and taking part in one of the family’s favorite rituals -- cheering for their beloved Philadelphia Eagles.



Doctors hope new technology like CRISPR gene-editing therapies can be the key to treating more than 7,000 rare diseases, such as sickle cell disease, cystic fibrosis, Huntington’s disease and muscular dystrophy, that affect approximately 30 million people across the country.



LINK: Baby saved by gene-editing therapy takes 1st steps ahead of Christmas
 
From ABC News:

The actual baby:
View attachment 943619

The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead of Christmas.

KJ Muldoon is walking and getting ready to celebrate the holiday season at home with his parents and three siblings.

KJ was born last year with a genetic disorder called carbamoyl-phosphate synthetase 1 deficiency. Also known as CPS1 deficiency, the metabolic condition affects only 1 in 1.3 million babies and often leads to life-threatening outcomes.

——-

Not in article but the wiki on disease if you are interested:
Carbamoyl phosphate synthetase I deficiency - Wikipedia

———

When he was 6 months old, doctors began giving KJ a groundbreaking new treatment -- a personalized CRISPR gene editing therapy at Children's Hospital of Philadelphia.

The gene-editing therapy works like a "molecular spell-checker,” finding and fixing the specific genetic error causing his condition.

After three infusions and after spending the first 10 months of his life in a hospital, KJ was dischargedand sent home in June.



Since then, KJ has continued to grow and thrive with his family, celebrating his first birthday at home and taking part in one of the family’s favorite rituals -- cheering for their beloved Philadelphia Eagles.



Doctors hope new technology like CRISPR gene-editing therapies can be the key to treating more than 7,000 rare diseases, such as sickle cell disease, cystic fibrosis, Huntington’s disease and muscular dystrophy, that affect approximately 30 million people across the country.



LINK: Baby saved by gene-editing therapy takes 1st steps ahead of Christmas
So he didn't died. So it's no Gore
 
Back
Top