Search Results - crispr

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Synthetic Exosomes (SE) for CNS Delivery of CRISPR for Gene Editing in Brain Disorders (UCLA Case No. 2021-363)

UCLA researchers from the Department of Neurology have developed synthetic exosomes, a novel delivery technology capable of transporting gene-editing tools and therapeutics across the blood-brain barrier, with successful applications in repairing Alzheimer’s-related mutations and delivering proteins, antibodies, and drugs. BACKGROUND: The emergence...

Published: 2/18/2025 | Inventor(s): Varghese John

Keywords(s): Alzheimers disease, CRISPR, delivery system, exosome, Nanotechnology, nanovesicles, synthetic biology

Category(s): Life Science Research Tools > Microfluidics And Mems, Materials > Nanotechnology, Platforms > Drug Delivery, Therapeutics > Gene Therapy And Editing, Therapeutics > CNS and Neurology

Identification of a Factor That Rescues Dysregulated Endocytosis in Cultured Human Hematopoietic Stem Cells (UCLA Case No. 2023-051)

UCLA researchers in the Departments of Molecular, Cell & Developmental Biology have developed a novel method to enhance the function of hematopoietic stem cells (HSCs) to improve the transplantability of ex vivo expanded human HSCs in clinical settings. BACKGROUND: Hematopoietic stem cells (HSCs), present in blood and bone marrow, can form mature...

Published: 2/14/2025 | Inventor(s): Hanna Mikkola, Julia Aguade Gorgorio

Keywords(s): bone marrow, bone marrow transplants, CRISPR, endocytosis, engraftment, environmental sensing, hematopoietic stem cells, leukemia, MLLT3, MYCT1, transplantability

Category(s): Therapeutics > Hematology, Life Science Research Tools, Therapeutics > Stem Cells And Regenerative Medicine

Targeted Epigenetic Editing as Novel Therapy for Malignant Glioma (UCLA Case No. 2023-047)

UCLA Researchers in the Department of Neurology/Neuro-Oncology and the David Geffen School of Medicine have developed methods for the Cas9-CRISPR mediated epigenetic targeting of malignant glioma, and which results in enhanced chemosensitivity to standard chemotherapeutic agents. BACKGROUND: Malignant gliomas are aggressive brain tumors originating...

Published: 2/14/2025 | Inventor(s): Serendipity Rinonos, Albert Lai

Keywords(s): Brain, Cancer, Cancer stem cells, Chemotherapy, Combination therapy, CRISPR, CRISPR-Cas9, Epigenetics, GBM, Glioma

Category(s): Therapeutics > Oncology, Therapeutics > CNS and Neurology

CRISPR Ribonucleoprotein Complex Genome Editing of Human Innate Immune Cells (UCLA Case No. 2021-056, Timothy O'Sullivan)

Researchers in the Department of Microbiology, Immunology, and Molecular Genetics at UCLA have developed an optimized strategy for non-viral CRISPR-Cas9 ribonucleoprotein gene editing of primary innate immune cells. BACKGROUND:CRISPR (clustered, regularly interspaced, short palindromic repeats) genome engineering has become a powerful tool to investigate...

Published: 2/14/2025 | Inventor(s): Timothy O'Sullivan, Luke Riggan, Andrew Hildreth

Keywords(s): CRISPR, Gene Editing Systems

Category(s): Therapeutics > Gene Therapy And Editing