{"id":242078,"date":"2026-08-02T21:06:36","date_gmt":"2026-08-03T02:06:36","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2026\/08\/gene-therapy-improves-stem-cell-transplant-success"},"modified":"2026-08-02T21:06:36","modified_gmt":"2026-08-03T02:06:36","slug":"gene-therapy-improves-stem-cell-transplant-success","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2026\/08\/gene-therapy-improves-stem-cell-transplant-success","title":{"rendered":"Gene Therapy Improves Stem Cell Transplant Success"},"content":{"rendered":"

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Stem cell transplantation (or bone marrow transplantation) and gene therapy have revolutionized the way oncologists treat patients. Both approaches have the potential to cure patients with sickle cell disease, b-thalassemia, immune disorders, and even several blood cancers. There are specific transplants that can occur: autologous and allogenic. Both forms of transplantation are dependent on the source of the donor cells. Autologous transplants use the patient\u2019s healthy stem cells taken from the patient before systemic treatment, like chemotherapy. Allogenic transplants use stem cells collected from healthy donors. Typically stems cells for an allogenic transplant is from a family member or umbilical cord blood to genetically match the recipient. If stem cells are not genetically matched, then the donor will recognize the stem cells as foreign and reject the transplant. This rejection is known as Graft-versus-Host Disease (GvHD). Infusion of stem cells only takes about 15 minutes, but the engraftment or the ability for cells to proliferate and establish themselves in the bone marrow takes up to 18 days. For full immune system recovery, most physicians see patients recover within a year. However, stem cell transplants are not perfect. There is a high rate of GvHD, infection, and disease relapse. Patients also usually receive chemotherapy before a transplant, which increases toxicity and reduces success of the procedure. Scientists are currently working on how to improve the success rate of transplantation.<\/p>\n

A recent article<\/a> in Nature<\/a>, by Dr. Pietro Genovese<\/a> and others, demonstrated that stem cell transplantations are safer when chemotherapy is replaced with a targeted treatment. Researchers used antibodies that recognize and target markers on blood-forming stem cells. This approach helps clear harmful preexisting stem cells from the patient before transplantation, instead of using toxic chemotherapeutic agents that damage DNA throughout the body. The antibody approach is more specific and reduces toxicity in patients. Genovese is an Assistant Professor at Dana Farber<\/a> and Boston Children\u2019s Cancer and Blood Disorders Center<\/a>. His work focuses on gene-editing and bioengineering technologies that improve stem and immune cells. Specifically, Genovese investigates ways to improve gene therapy and develop approaches to enhance treatment for patients with hematological malignancies.<\/p>\n

Previously, an antibody could not discern between current recipient and infused autologous stem cells. Antibodies could also attack transplanted cells from a separate donor (allogenic). To avoid these issues, Genovese and his team have used gene-editing tools to select for a specific biomarker on the surface of donor stem cells. The small edit prevented the antibody from binding to the donor stem cells. These antibodies were able to avoid the healthy stem cells and still eliminate the infected stem cells. This approach allows the donor stem cells to properly graft in the host and improve success of transplantation.<\/p>\n","protected":false},"excerpt":{"rendered":"

Stem cell transplantation (or bone marrow transplantation) and gene therapy have revolutionized the way oncologists treat patients. Both approaches have the potential to cure patients with sickle cell disease, b-thalassemia, immune disorders, and even several blood cancers. There are specific transplants that can occur: autologous and allogenic. Both forms of transplantation are dependent on the [\u2026]<\/p>\n","protected":false},"author":662,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1902,11,412],"tags":[],"class_list":["post-242078","post","type-post","status-publish","format-standard","hentry","category-bioengineering","category-biotech-medical","category-genetics"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/242078","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/662"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=242078"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/242078\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=242078"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=242078"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=242078"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}