![]() If you are in a biotech or pharmaceutical company, and would like a 15-20-minute, no-obligation telephone discussion of issues raised by this or other blog articles, or of other issues that are important to your company, please contact us by phone or e-mail. However, they will still need to be monitored long-term to determine if the treatment is durable and free of side effects over the long term.Īs the producers of this blog, and as consultants to the biotechnology and pharmaceutical industry, Haberman Associates would like to hear from you. Children treated with this gene therapy should therefore be protected against common ailments by their reconstituted immune systems. The researchers concluded that the IL2RG-lentiviral vector gene therapy combined with low-exposure, targeted busulfan conditioning in infants with newly diagnosed SCID-X1 showed low-grade acute toxic effects, and resulted in engraftment of transduced cells, reconstitution of functional T cells and B cells, and normalization of NK-cell counts during a median follow-up of 16 months. The subjects also showed other signs of immune system normalization, including vaccine response in three of the infants. Previous infections were cleared in all infants, and all continued to grow normally. However, a boost of gene-corrected cells without busulfan conditioning resulted in T-cell normalization. The eighth subject initially had an insufficient T-cell count. The vector was present in T cells, B cells, NK cells, myeloid cells, and bone marrow progenitors in these seven subjects. In seven of the infants, the numbers of T cells and NK cells normalized by 3 to 4 months after infusion. The eight infants were studied for a median of 16.4 months, and experienced no unexpected side effects. ( “Conditioning”, for example via a myelosuppressive chemotherapy like busulfan given prior to stem-cell transplantation, is designed to make room for transplanted blood stem cells to grow. ![]() The transfected stem cells were infused back into eight infants with newly diagnosed SCID-X1after low-exposure, targeted busulfan conditioning. This was used to transfect patient-derived bone-marrow stem cells. The new experimental gene therapy for SCID-X1 utilized a lentiviral vector carrying IL2RG complementary DNA. We discussed the development and use of lentiviral vectors in our November 2015 book-length report, Gene Therapy: Moving Toward Commercialization, published by Cambridge Healthtech Institute. Instead, researchers have developed lentiviral vectors, which appear to have a lower risk of leukemogenesis than gammaretroviral vectors. This resulted in a high level of leukemia induction, as discussed in a previous article on this blog. However, fewer than 20% of SCID-X1 patients have such an available donor.Ī previous attempt to apply gene therapy to treatment of SCID-X1, in the early 2000s, utilized a Moloney murine leukemia virus (MoMuLV) gammaretrovirus as a vector. SCID-X1 can sometimes be cured by a bone-marrow transplant from a matched sibling donor. It probably affects at least 1 in 50,000 to 100,000 newborns. As a result, they are very susceptible to infections, and typically die before age 2 if not isolated or treated.Īlthough SCID-X1 is a rare disease, it is the most common form of severe combined immunodeficiency. Individuals with SCID-X1 produce very few T and NK (natural killer) cells, and are thus severely immunodeficient. This gene encodes the interleukin-2 receptor subunit gamma (IL-2RG), which is common to the receptor complexes for at least six different interleukin receptors, including IL-2 and IL-4. SCID-X1 is a rare X-linked genetic disease caused by a mutation in the L2RG gene. ![]() SCID-X1 is sometimes called “bubble-boy disease”, because of the case of a boy born in 1971 with SCID-X1, who had to be isolated in a plastic bubble while awaiting a bone-marrow transplant. Jude Children’s Research Hospital (Memphis, TN) and their colleagues have used gene therapy to restore immune function to eight infants with newly diagnosed X-linked severe combined immunodeficiency (SCID-X1). ()Īs reported in the 18 April issue of the New England Journal of Medicine, researchers at the St. IL2RG protein, encoded by tL2RG complementary DNA.
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