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While progress in gene therapy for red-green color blindness has slowed since then, successful human trials are currently underway for achromatopsia, a different form of color vision deficiency. Congenital color vision deficiency affects over 200 million people worldwide, highlighting the significant demand for effective gene therapies ...
Stem-cell therapy uses stem cells to treat or prevent a disease or condition. [1] As of 2024, the only FDA-approved therapy using stem cells is hematopoietic stem cell transplantation. [2] [3] This usually takes the form of a bone marrow or peripheral blood stem cell transplantation, but the cells can also be derived from umbilical cord blood.
It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in May 2018. [5] Elivaldogene autotemcel is made specifically for each recipient, using the recipient's hematopoietic stem cells. [6] It was approved for medical use by the U.S. Food and Drug Administration in September 2022 ...
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For over 90 years, hematopoietic stem cell transplantation (HSCT) has been used to treat people with conditions such as leukaemia and lymphoma; this is the only widely practiced form of stem-cell therapy. [94] [97] [98] As of 2016, the only established therapy using stem cells is hematopoietic stem cell transplantation. [99]
A stem cell line is a group of stem cells that is cultured in vitro and can be propagated indefinitely. Stem cell lines are derived from either animal or human tissues and come from one of three sources: embryonic stem cells, adult stem cells, or induced pluripotent stem cells. They are commonly used in research and regenerative medicine.
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