Gene Therapy for Leber Congenital Amaurosis
The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of an AAV gene therapy for Leber Congenital Amaurosis caused by CEP290 mutations.
RS-1 Knockout Mouse Model for X-Linked Retinoschisis (XLRS) Research
The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of a transgenic mouse model for X-linked retinoschisis (XLRS).
Generating Conditional and Reverse Conditional Loss-of-Function Alleles in Mouse Casq2
Summary:
The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) is seeking potential licensees interested in further developing or utilizing these Casq2 mouse strains. As a research tool, patent protection is not being pursued for this technology. More information to access these strains can be found here: https://www.jax.org/strain/036291 and https://www.jax.org/strain/036290.
Genetically Modified Traf3ip2-/- Mice as a Valuable Resource for Exploring IL-17 Signaling in Autoimmune, Inflammatory Diseases, and Beyond
Traf3ip2-/- C57/BL6 mice are a genetically modified mouse model in which the Traf3ip2 gene, responsible for encoding the CIKS adaptor protein essential for IL-17 cytokine signaling, has been disrupted. These mice offer a robust platform for research in autoimmune and inflammatory diseases, as well as potential applications in cancer studies. By eliminating IL-17 signaling and cross-interactions with other pathways, they provide a unique opportunity for drug discovery and proof-of-principle studies, shedding light on disease mechanisms and therapeutic development.
Comprehensive Examination of Nuclear Envelope Defects Through a Rabbit Polyclonal Antibody Targeting Human Sun1 Inner Nuclear Membrane Protein
The technology at hand involves a rabbit polyclonal antibody specifically designed for the human Sun1 inner nuclear envelope protein, even though it is directed against the mouse Sun1 inner nuclear membrane protein. Sun1 is known to be an inner nuclear envelope protein, and defects in such proteins can lead to debilitating conditions like Emery-Dreifuss muscular dystrophy and Hutchinson Gilford Progeria Syndrome. Importantly, the antibody serves as a valuable tool for diagnostic and analytical studies concerning cells afflicted with nuclear envelope defects.
Development of a Novel mRNA-Based Immunization Strategy for Inducing Protective Immunity to HIV
This innovative HIV immunization strategy relies on mRNA technology to prime and boost immune responses, using full-length or minimally truncated Env proteins to maintain native conformation for effective membrane expression. Co-formulating Env and Gag proteins promotes virus-like particle (VLP) production in vivo, mimicking HIV's structure for better immune recognition. The regimen involves an intensive schedule of 8+ sequential immunizations and includes boosting with diverse Envs from different HIV-1 clades to broaden the immune response.
Isolating Potent CD8+ T Cell Receptors from HIV Controllers for Advanced Therapies
Novel CD8+ T cell receptors (TCRs) isolated from elite HIV controllers show unprecedented affinity for conserved HIV epitopes, offering new avenues for direct immunotherapies and T cell engineering. These TCRs exhibit potential for cytotoxicity mediation against HIV-infected cells, with prospects for use in toxin-coupled treatments or as part of engineered T cell therapies. Collaborations, such as with Altor Biosciences, are enhancing these TCRs with proprietary molecules like IL-15, to bolster therapeutic efficacy.
Advancements in Hematopoietic Stem Cell Transplantation: Non-Toxic Conditioning with CD117-Targeted Monoclonal Antibodies
The technology revolves around an innovative monoclonal antibody-based conditioning regimen for enhancing the engraftment of hematopoietic stem cells during bone marrow transplants. It features a novel antibody-drug conjugate that targets CD117, a marker on stem cells, to enable effective transplantation without the harmful side effects of traditional conditioning methods.
Synthetic Peptide Immunogens for Broadly Neutralizing Antibody Induction Against HIV-1
The technology in focus encompasses a novel suite of synthetic peptide immunogens, collaboratively developed by leading institutions, aimed at evoking a robust immune response against HIV-1. This breakthrough harnesses the latest advancements in immunology to craft immunogens that elicit broadly neutralizing antibodies in humans, a significant stride in HIV-1 therapeutic and preventive strategies.
Self-Assembling Nanoparticle System for Scalable and Potent Individualized Cancer Vaccines
The technology pertains to a novel polymer nanoparticle platform designed for the creation of individualized cancer vaccines. It utilizes a two-component system that combines patient-specific peptide neoantigens with immunostimulants within self-assembling nanoparticles. This approach ensures the targeted delivery of neoantigens to the immune system, enhancing the immune response against cancer cells while avoiding the systemic activation often seen with conventional adjuvants.