Search Results - therapeutics+%3e+stem+cells+and+regenerative+medicine

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Vectors Combining Anti-Sickling Beta-AS3-Globin with Anti BCL11A ShRNAmir to Treat Beta-Hemoglobinopathies (UCLA Case No. 2021-190)

UCLA researchers from the Department of Microbiology, Immunology and Molecular Genetics and Pediatrics have developed a potent bi-functional lentiviral expression vector to treat sickle cell disease and beta-thalassemia. BACKGROUND: Beta-hemoglobinopathies, such as sickle cell disease and beta-thalassemia, are genetic disorders characterized by dysfunctional...

Published: 7/17/2025 | Inventor(s): Donald Kohn

Keywords(s):

Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Hematology, Therapeutics > Stem Cells And Regenerative Medicine

Small Molecule Enhancers of Secreted Clusterin (sCLU) as Novel Therapeutics for Alzheimer's Disease (UCLA Case No. 2024-016)

UCLA researchers from the Department of Neurology have identified and synthesized novel small molecules that enhance the levels of secreted clusterin protein, a protein that can targets a significant risk factor for the disease, and offering promising potential for the treatment of Alzheimer’s disease. BACKGROUND: Alzheimer’s disease (AD)...

Published: 7/17/2025 | Inventor(s): Varghese John

Keywords(s):

Category(s): Therapeutics > CNS and Neurology, Therapeutics > Stem Cells And Regenerative Medicine

Gene Therapy for DOCK8 Deficiency (UCLA Case No. 2023-178)

UCLA researchers in the Department of Pediatrics Allergy and Immunology have developed a gene therapy method to express the full-length DOCK8 protein in cells, aimed at treating DOCK8 deficiency. These method allows for safe and effective DOCK8 expression in hematopoietic stem cells without the need for an HLA-matched donor, enabling patients to undergo...

Published: 7/17/2025 | Inventor(s): Caroline Kuo

Keywords(s): bone marrow, bone marrow transplants, DOCK8, DOCK8 deficiency, Gene Editing Systems, Gene Therapy, hematopoietic stem cells, Immune System, immunodeficiency, Immunology, intein splicing

Category(s): Therapeutics > Immunology And Immunotherapy, Therapeutics > Gene Therapy And Editing, Therapeutics > Stem Cells And Regenerative Medicine, Life Science Research Tools > Plasmids/Vectors

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: 7/17/2025 | 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

A Novel “Host-Guest”-Based Supramolecular Hydrogel for Dental Pulp Regeneration (Case No. 2023-154)

Summary: Researchers in the Department of Dentistry at UCLA have developed an innovative hydrogel for a vast array of biomedical and tissue engineering applications. Background: Hydrogels have emerged as powerful tools in biomedical engineering due to their ability to provide a supportive environment for cell growth, tissue repair, and drug delivery....

Published: 4/29/2025 | Inventor(s): Alireza Moshaverinia, Weihao Yuan

Keywords(s): anti-dehydration, Drug Delivery, dynamic identification, hydrogel, Stem Cells / Regenerative Medicine, Targeted Therapy, tissue regeneration

Category(s): Chemical, Materials > Functional Materials, Platforms > Drug Delivery, Platforms > Drug Delivery > Antibody Delivery Platforms, Platforms > Drug Delivery > Nanoparticles, Platforms > Drug Delivery > Viral Delivery Systems, Therapeutics > Cell Therapy, Therapeutics > Medication, Therapeutics > Stem Cells And Regenerative Medicine

Compounds and Methods for Treating Cancer by Destroying Cancer Stem Cells (UCLA Case No. 2021-286)

Researchers from UCLA’s Department of Chemistry and Biochemistry and the Department of Radiation Oncology have synthesized a set of compounds for the eradication of cancer stem cells. These compounds are predicted to cross the blood brain barrier and show promise as effective interventions for glioblastoma in both in vitro and in vivo assays. BACKGROUND:...

Published: 7/17/2025 | Inventor(s): Michael Jung, Frank Pajonk

Keywords(s): blood-brain barrier, Brain, Cancer, Cancer stem cells, drug screening, GBM, Glioblastoma, Glioma, new chemical entity

Category(s): Therapeutics > Oncology, Therapeutics > CNS and Neurology, Therapeutics > Stem Cells And Regenerative Medicine

Method for Combined Conditioning and Chemoselection in a Single Cycle (UCLA Case No. 2011-478)

SUMMARY: Researchers at UCLA have developed a highly-efficient technique to engraft and select hematopoietic stem cells directly inside a patient. The invention will improve the fidelity of stem cell transplantations and reduce toxic side effects on patients. BACKGROUND: Hematopoietic stem cell transplantation (HSCT) is a mainstay of treatment for...

Published: 7/17/2025 | Inventor(s): Gay Crooks, Robert Schiestl, Katrin Hacke, Akos Szakmary

Keywords(s): blood cancers, Cancer stem cells, Hematopoietic stem cell transplantation, hematopoietic stem cells, lymphatic cancer, stem cell transplantations, Transplant

Category(s): Therapeutics > Stem Cells And Regenerative Medicine, Therapeutics > Transplant

Combining Autologous or Allogeneic Total Monocytes or CD16+ Monocytes to Protect and Increase the Expansion and Function of Stem Cells (UCLA Case No. 2021-182)

SUMMARY: UCLA researchers in the department of Dentistry have developed a novel approach to increase the successful transplantation and survival of stem cells into patients. BACKGROUND: The World Health Organization has recognized that transplantation of human cells saves and restores essential life functions to patients who are not presented with...

Published: 7/17/2025 | Inventor(s): Anahid Jewett

Keywords(s):

Category(s): Therapeutics, Therapeutics > CNS and Neurology, Therapeutics > Immunology And Immunotherapy, Therapeutics > Stem Cells And Regenerative Medicine, Therapeutics > Stem Cells And Regenerative Medicine > Ips Stem Cells

2019-870 GENE EDITING OF MONOGENIC DISORDERS IN HUMAN HEMATOPOIETIC STEM CELLS (XHIM)

Researchers at the UCLA Department of Microbiology, Immunology & Molecular Genetics have developed novel methods to achieve efficient, precise gene integration and effective expression of cDNA cassettes to express normal versions of genes in hematopoietic stem cells. BACKGROUND:Hematopoietic stem cells (HSCs)—stem cells that give rise to...

Published: 7/17/2025 | Inventor(s): Donald Kohn

Keywords(s): Gene Editing Systems, Gene Therapy, Genetic Disease, Hematology, Immunotherapy, iPS/stem cells, Therapeutics & Vaccines, Viral Delivery Systems

Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Hematology, Therapeutics > Immunology And Immunotherapy, Therapeutics > Stem Cells And Regenerative Medicine, Therapeutics > Stem Cells And Regenerative Medicine > Ips Stem Cells

2020-443 A PATCH OF DETACHABLE HYBRID MICRONEEDLE DEPOT (D-HMND) FOR LOCALIZED DELIVERY OF MESENCHYMAL STEM CELLS IN REGENERATION THERAPY

UCLA researchers in the Department of Bioengineering have developed a novel detachable hybrid microneedle depot (d-HMND) for the targeted delivery of mesenchymal stem cells. BACKGROUND: Stem cell biology has been widely applied to the treatment of tissue damage, due to the ability of stem cells to form, repair, and maintain tissues and organs after...

Published: 7/17/2025 | Inventor(s): Ali Khademhosseini

Keywords(s): iPS/stem cells, Life Science Research Tools, Stem Cells / Regenerative Medicine, Therapeutics & Vaccines

Category(s): Therapeutics > Stem Cells And Regenerative Medicine, Therapeutics > Stem Cells And Regenerative Medicine > Ips Stem Cells

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