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Gene Therapy Cures Inherited Diseases

Technology

2025-07-23

2:00 PM

786

Published by BBC News

Gene Therapy Cures Inherited Diseases

Medical researchers have achieved remarkable success in using gene therapy to cure several inherited diseases that were previously considered incurable. The breakthrough treatments use advanced CRISPR gene editing technology to correct genetic defects at the cellular level. Clinical trials have shown complete remission in patients with conditions such as sickle cell disease, muscular dystrophy, and certain forms of blindness. The gene therapy approach involves modifying patients' own cells to correct genetic mutations, eliminating the need for lifelong treatments or organ transplants. Regulatory agencies have fast-tracked approval for several gene therapies based on their exceptional safety and efficacy profiles. The success of these treatments is opening new possibilities for addressing thousands of rare genetic disorders that affect millions of people worldwide. However, the high cost of gene therapy remains a challenge, prompting discussions about healthcare accessibility and insurance coverage.

Ravi

Great article!

Sneha

Very informative. Thanks!

Amit

I learned a lot from this.

Gene Therapy Cures Inherited Diseases

Technology

2025-07-23

2:00 PM

786

Published by BBC News

Gene Therapy Cures Inherited Diseases

Medical researchers have achieved remarkable success in using gene therapy to cure several inherited diseases that were previously considered incurable. The breakthrough treatments use advanced CRISPR gene editing technology to correct genetic defects at the cellular level. Clinical trials have shown complete remission in patients with conditions such as sickle cell disease, muscular dystrophy, and certain forms of blindness. The gene therapy approach involves modifying patients' own cells to correct genetic mutations, eliminating the need for lifelong treatments or organ transplants. Regulatory agencies have fast-tracked approval for several gene therapies based on their exceptional safety and efficacy profiles. The success of these treatments is opening new possibilities for addressing thousands of rare genetic disorders that affect millions of people worldwide. However, the high cost of gene therapy remains a challenge, prompting discussions about healthcare accessibility and insurance coverage.

Ravi

Great article!

Sneha

Very informative. Thanks!

Amit

I learned a lot from this.