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In 1992, Claudio Bordignon, working at the Vita-Salute San Raffaele University, performed the first gene therapy procedure using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases. [169] In 2002, this work led to the publication of the first successful gene therapy treatment for ADA-SCID.
The Food and Drug Administration on Friday approved Pfizer’s treatment for a rare genetic bleeding disorder, making it the company’s first-ever gene therapy to win clearance in the U.S.
With the discovery of various types of immune-related disorders, there is a need for diversification in prevention and treatment. Developments in the field of gene therapy are being studied to be included in the scope of this treatment, but of course more research is needed to increase the positive results and minimize the negative effects of gene therapy applications. [27]
Gene delivery is a necessary step in gene therapy for the introduction or silencing of a gene to promote a therapeutic outcome in patients and also has applications in the genetic modification of crops. There are many different methods of gene delivery for various types of cells and tissues.
Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available); Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1]
A more recent focus of the journal includes Cell Therapy, with genetic modification, in essence ex vivo gene therapy. Previous issues have focused on the latest developments in gene transfer, gene expression and regulation, development of novel gene delivery vectors, and ex vivo gene therapies, animal models, and human therapeutic applications.
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TIL that Gene Roddenberry originally did not want to cast Patrick Stewart as Picard, since he had envisioned an actor who was "masculine, virile, and had a lot of hair". Image credits ...