Researchers have identified an early postnatal window that allows gene transfer to circulating blood stem cells, advancing ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

Two groundbreaking biotechnology advances are fundamentally altering human health and food production in 2025, moving from ...

With gene therapy offering new hope for thalassemia, experts urge Indian govt to invest in early diagnosis, genetic testing, ...

Cell and gene therapy (C&GT) is set to revolutionise treatment of complex diseases, offering new possibilities for patients.

Thalassemia, a group of inherited blood disorders characterized by reduced or abnormal hemoglobin production, continues to ...

In medical breakthrough, a baby diagnosed with rare genetic disorder is thriving after customized CRISPR gene editing therapy ...

China announced Wednesday that four children with thalassemia, a severe genetic blood disorder usually diagnosed in toddlers, have been cured using a domestically developed DNA base editing therapy ..

A new gene therapy developed at the Children's Hospital ... Nabeel was born with an inherited blood disorder called beta thalassemia. "It was just, like, so painful," she said.

A stem cell transplant and gene therapy may also be options ... symptoms related to moderate or severe anemia. With beta thalassemia major, you may have more symptoms. They may be severe.

The development and approval of gene therapies has been a potential game changer for patients with beta-thalassaemia ... eligibility for gene therapy for thalassemia comes down to two main ...