Search
22 articles for “regulatory condition”
-
Gene Therapy in Modern Medicine: Promises and Challenge in Treating Genetic Diseases
Abstract: Gene therapy is a medical approach that focuses on altering or adjusting an individual’s genes to treat or prevent illnesses. The aim is to repair faulty genes or insert new ones into the body to combat diseases. Gene therapy can involve directly introducing modified or new genes into a patient’s cells or altering the genes already present in the patient’s body. This approach shows potential for treating a range of …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 2, Issue 2, 2024 · pp. 27–32 Read article
-
Prime Editing and Base Editing in Human Hematopoietic Stem Cells Toward Scarless Correction of Monogenic Blood Disorders
Abstract: Monogenic hematological disorders – such as sickle cell disease (SCD), β-thalassemia, and X-linked chronic granulomatous disease (X-CGD) – affect >400, 000 newborns worldwide each year, with a considerable burden in low-resource settings . Although donor-derived allogeneic hematopoietic stem-cell transplantation (HSCT) is widely regarded as curative, its application is limited due to issues of donor availability and graft-versus-host disease (GVHD) and conditioning-related toxicity. DSB-based conventional CRISPR-Cas9 strategies are limited by inefficient …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 4, Issue 1, 2026 Read article