"One attempt at modifying human DNA was performed in 1980, by Martin Cline."
The treatment of genetic disorders by inserting, deleting or correcting specific genes to restore normal function.
"The first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989."
"The first therapeutic use of gene transfer was performed by French Anderson in a trial starting in September 1990."
"Between 1989 and December 2018, over 2,900 clinical trials were conducted."
"More than half of them [clinical trials] were in phase I."
"In 2003, Gendicine became the first gene therapy to receive regulatory approval."
"Further gene therapy drugs were approved, such as Glybera (2012), Strimvelis (2016), Kymriah (2017), Luxturna (2017), Onpattro (2018), Zolgensma (2019), Abecma (2021), Adstiladrin, Roctavian and Hemgenix (all 2022)."
"Most of these approaches utilize adeno-associated viruses (AAVs) and lentiviruses for performing gene insertions."
"AAVs are characterized by stabilizing the viral capsid, lower immunogenicity, ability to transduce both dividing and nondividing cells, the potential to integrate site specifically and to achieve long-term expression in the in-vivo treatment."
"ASO/siRNA approaches such as those conducted by Alnylam and Ionis Pharmaceuticals require non-viral delivery systems."
"ASO/siRNA approaches...utilize alternative mechanisms for trafficking to liver cells by way of GalNAc transporters."
"Bone marrow transplantation and organ transplants in general have been found to introduce foreign DNA into patients."