Genetic Therapies - What Are Genetic Therapies? | NHLBI, NIH
Mar 24, 2022 · These changes can cause genetic, or inherited, disorders such as cystic fibrosis, thalassemia, hemophilia, and sickle cell disease. Genetic therapies are approaches that treat genetic …
How are genetic conditions treated or managed? - MedlinePlus
May 2, 2021 · Most treatment strategies for genetic disorders do not alter the underlying genetic mutation; however, a few disorders have been treated with gene therapy. This experimental …
The Rarest of All Diseases Are Becoming Treatable
3 days ago · The Rarest of All Diseases Are Becoming Treatable This year, gene-editing technology was customized to fix mutations in a single patient’s genes for the first time.
Gene therapy - Mayo Clinic
Apr 23, 2024 · Gene therapy aims to fix a faulty gene or replace it with a healthy gene to try to cure disease or make the body better able to fight disease. It holds promise as a treatment for a wide …
How Gene Therapy Can Cure or Treat Diseases | FDA
Jul 28, 2022 · Advancements in science are changing how we define disease, develop drugs, and prescribe treatments. FDA has approved gene therapies for cancer, rare diseases.
Findings point to path forward for treatment of rare genetic disease
2 days ago · There is no widely approved treatment that modifies the disease, and existing therapies may not be effective for all patients. Researchers from Mass General Brigham and the Broad …
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease
May 15, 2025 · Base editors can correct disease-causing genetic variants. After a neonate had received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with an estimated …
What Is Gene Therapy? Pros, Cons & Examples - Cleveland Clinic
Jun 17, 2025 · What is gene therapy? Gene therapy is a type of treatment for genetic disorders. It works by either changing a disease-causing gene or giving you a working copy of that gene. Genes are …
What is a Genetic Disorder? Understanding Causes, Types, and Treatments
Apr 18, 2025 · Gene therapy involves introducing, removing, or altering genetic material within a person’s cells to treat or prevent disease. While gene therapy is still in the experimental stages for …
New Artificial Intelligence Model Could Speed Rare Disease Diagnosis
Nov 24, 2025 · At a glance: A new AI model called popEVE can predict how likely each variant in a patient’s genome is to cause disease. The team is testing popEVE in clinical settings to see if it can …