Gene therapy - Mayo Clinic
2024年4月23日 · 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 range of diseases, such as cancer, cystic fibrosis, …
The once and future gene therapy | Nature Communications
2020年11月16日 · Gene therapy is at an inflection point. Recent successes in genetic medicine have paved the path for a broader second wave of therapies and laid the foundation for next-generation...
The Definition of Gene Therapy Has Changed - Nature
2021年10月26日 · Today, because of that progress, many gene therapies employ adeno-associated virus (AAV) or retrovirus vectors, each with their own pros and cons, in addition to improved versions of the ...
The Current Status of Gene Therapy for the Treatment of Cancer
Gene therapy for cancer treatment has good progress in the last three decades, few drugs approved, while others are still in trials. Relatively gene therapy has better safety with tolerable adverse effects than chemotherapy for the treatment of cancer.
Gene therapy - Latest research and news - Nature
2024年10月31日 · Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA...
A new age of precision gene therapy - The Lancet
2023年11月23日 · Gene therapy has become a clinical reality as market-approved advanced therapy medicinal products for the treatment of distinct monogenetic diseases and B-cell malignancies. This Therapeutic Review aims to explain how progress in genome editing technologies offers the possibility to expand both therapeutic options and the types of …
The state of cell and gene therapy in 2023 - Cell Press
Gene, cell, and RNA therapies, collectively called cell and gene therapies (CGTs) in this review, represent the cutting edge of drug discovery (Figure 1). Owing to their precise nature, targeted against the genetic and molecular drivers of disease, these therapeutic approaches are the embodiment of precision medicine.
Gene Therapy News - ScienceDaily
2 天之前 · Latest Headlines. updated 7:56pm EST. Effective New Gene Therapy to Treat Multiple Sulfatase Deficiency. Sep. 9, 2024 — Researchers tested a preclinical model for an experimental gene...
FDA approves first gene-editing treatment for human illness
2023年12月8日 · The Food and Drug Administration approved two genetic treatments for sickle cell disease, including one that uses gene-editing. The approvals offer hope for patients and signal a new medical era...
The world's 1st CRISPR therapy has been approved. Here's …
2024年1月18日 · The world's first treatment that uses CRISPR gene-editing technology has been approved. Exa-cel, also known by its brand name Casgevy, received its first regulatory approval on Nov. 16, 2023 from...