{"id":383557,"date":"2026-04-17T03:14:07","date_gmt":"2026-04-17T03:14:07","guid":{"rendered":"https:\/\/www.newsbeep.com\/nz\/383557\/"},"modified":"2026-04-17T03:14:07","modified_gmt":"2026-04-17T03:14:07","slug":"stem-cell-editing-programs-the-immune-system-to-make-own-therapeutic-proteins","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/nz\/383557\/","title":{"rendered":"Stem Cell Editing Programs the Immune System to Make Own Therapeutic Proteins"},"content":{"rendered":"

\"Dividing<\/a>Credit: JUAN GAERTNER\/ Science Photo Library \/ Getty Images<\/p>\n

For pathogens like HIV, malaria, and rapidly evolving influenza strains, coaxing the immune system to produce the rare, highly potent antibodies needed for protection has long been a scientific bottleneck. Vaccines can train B cells to evolve such broadly neutralizing antibodies<\/a>, but only under ideal conditions\u2014and only in a small fraction of people. Even attempts to genetically edit mature B cells produced responses that faded as the cells died out.<\/p>\n

A team at the Rockefeller University has now taken a more upstream approach: programming hematopoietic stem and progenitor cells (HSPCs)\u2014the source of all B lymphocytes\u2014to carry permanent genetic instructions for therapeutic antibodies or other proteins. Because the immune system naturally amplifies rare, useful cells after vaccination, even a tiny number of edited stem cells can seed a durable, boostable immune response.<\/p>\n

\u201cThe immune system is inefficient in that it produces a vast quantity of cells to protect itself,\u201d said Harald Hartweger, a research assistant professor in Michel Nussenzweig\u2019s Laboratory of Molecular Immunology. \u201cWe wanted to take advantage of the immune system\u2019s ability to amplify useful, rare cells.\u201d<\/p>\n

The study, published in Science and titled \u201cB lymphocyte protein factories produced by hematopoietic stem cell gene editing<\/a>,\u201d demonstrates that CRISPR\u2011edited HSPCs can mature into B cells that express engineered antibodies upon vaccination. A standard vaccination then acts as the trigger: antigen exposure drives those edited B cells to expand, differentiate into plasma cells, and secrete high titers of the inserted antibody that last long-term.<\/p>\n

According to the paper, as few as ~7,000 edited HSPCs were enough to generate \u201chigh titers of long\u2011lasting protective or therapeutic antibodies and\/or cargo proteins.\u201d In mice engineered to produce a broadly neutralizing influenza antibody, this response was strong enough to protect against an otherwise lethal viral infection.<\/p>\n

The platform proved unexpectedly versatile. Edited B cells could also secrete non\u2011antibody proteins, pointing to potential applications in genetic diseases. And by mixing HSPCs engineered with different antibody instructions, the researchers created immune systems capable of producing multiple antibodies simultaneously, an approach that could limit viral escape in HIV or other rapidly mutating pathogens. Human HSPCs edited using the same strategy produced functional human B cells in an immunodeficient mouse model, offering an early sign of translational feasibility.<\/p>\n

\u201cOur goal is to permanently impact the genome with a single injection, so that the body can make proteins of interest,\u201d Hartweger said. \u201cThat protein could be an antibody that\u2019s universally protective against HIV or influenza, but it could also be any therapeutic protein.\u201d<\/p>\n

The team is now moving toward preclinical testing in non\u2011human primates to evaluate protection against HIV and exploring whether similar strategies could be applied to T cells. The broader vision is a generalizable, long\u2011term protein\u2011production platform, one that could support treatments for infectious disease, protein deficiencies, autoimmunity, metabolic disorders, and cancer, according to Hartweger.<\/p>\n

As Nussenzweig puts it, \u201cThe present study proposes a workaround for the antibody problem\u2014a way of getting around the possibility that we may never get to a universal HIV vaccine, while still providing a promising, long\u2011lasting solution.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"Credit: JUAN GAERTNER\/ Science Photo Library \/ Getty Images For pathogens like HIV, malaria, and rapidly evolving influenza…\n","protected":false},"author":2,"featured_media":383558,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[10],"tags":[27759,134,111,43,139,69,2477,16227],"class_list":{"0":"post-383557","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-health","8":"tag-genome-editing","9":"tag-health","10":"tag-new-zealand","11":"tag-news","12":"tag-newzealand","13":"tag-nz","14":"tag-topics","15":"tag-translational-medicine"},"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/posts\/383557","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/comments?post=383557"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/posts\/383557\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/media\/383558"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/media?parent=383557"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/categories?post=383557"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/tags?post=383557"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}