UCLA Researchers & Innovators
Industry & Investors
News & Events
About
Concierge
Search Results - genetic+disease
16
Results
Sort By:
Published Date
Updated Date
Title
ID
Descending
Ascending
Gene Augmentation Therapy for Usher Syndrome 1B (UCLA Case No. 2023-138)
UCLA researchers in the Department of Ophthalmology have developed a novel approach to cure Usher Syndrome 1B. BACKGROUND: Usher syndrome is defined as a condition whose major symptoms are deafness or hearing loss, and an eye disease called retinitis pigmentosa (RP), which affects vision. The ear-related effects of Usher’s disease are caused...
Published: 2/14/2025
|
Inventor(s):
David Williams
Keywords(s):
Gene delivery
,
Genetic Disease
,
Hearing loss
,
Usher syndrome
,
Vision loss
Category(s):
Therapeutics > Gene Therapy And Editing
Optimized Lentiviral Vector for Stem Cell Gene Therapy of Hemoglobinopathies
UCLA Case No. 2017-460 Summary UCLA researchers in the Department of Microbiology, Immunology and Molecular Genetics have developed a novel method to produce short lentiviral vectors with tissue-specific expression, with a primary focus on lentiviral vectors for treating sickle cell disease and other disorders of hemoglobin. Background Sickle...
Published: 2/14/2025
|
Inventor(s):
Donald Kohn
,
Roger Hollis
,
Richard Morgan
Keywords(s):
Drug Delivery
,
Gene Editing Systems
,
Gene Therapy
,
Genetic Disease
,
Life Science Research Tools
,
Plasmids / Vectors
,
Research Methods
,
Therapeutics & Vaccines
,
Viral Delivery Systems
Category(s):
Life Science Research Tools > Plasmids/Vectors
,
Life Science Research Tools
,
Life Science Research Tools > Research Methods
,
Platforms > Drug Delivery
,
Therapeutics
,
Platforms > Drug Delivery > Viral Delivery Systems
,
Therapeutics > Gene Therapy And Editing
2020-777: Correcting Neuronal Dysfunction in Intellectual Disability Syndromes
UCLA researchers in the department of Molecular, Cellular, and Developmental Biology have developed a treatment that could mitigate some mental issues related to various intellectual disabilities syndromes. BACKGROUND: Approximately 6.5 million people in the United states have an intellectual disability syndrome, classified as an IQ of less than 70....
Published: 2/14/2025
|
Inventor(s):
William Lowry
Keywords(s):
Cns
,
Genetic Disease
,
Therapeutics & Vaccines
Category(s):
Therapeutics
,
Therapeutics > CNS and Neurology
2019-720: Preventing Senescence to Improve Neuronal Function in Intellectual Disability Disorders
UCLA researchers in the Department of Molecular and Cell & Developmental Biology have developed novel therapeutic strategy to treat intellectual disability disorders discovered via a screening platform for drug discovery in a human model of brain development. BACKGROUND: Rett syndrome is an X-linked genetic neurological disease associated...
Published: 2/14/2025
|
Inventor(s):
William Lowry
Keywords(s):
Cns
,
Genetic Disease
,
Therapeutics & Vaccines
Category(s):
Therapeutics > CNS and Neurology
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: 2/14/2025
|
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
2020-428 CILENGITIDE AS A NEW AGENT FOR THE TREATMENT OF DYSREGULATED WOUND HEALING IN EHLERS DANLOS SYNDROME
UCLA researchers in the Department of Medicine have uncovered a new use of cilengitide for the treatment of dysregulated wound healing in Classic Ehlers-Danlos syndrome.BACKGROUND:Classic Ehlers-Danlos syndrome (cEDS) is a
genetic disease
that weakens the connective tissues in the body. Mutations in the Col5a1 or Col5a2 genes mean that production of...
Published: 2/14/2025
|
Inventor(s):
Arjun Deb
Keywords(s):
Cardiovascular
,
Genetic Disease
,
Therapeutics & Vaccines
Category(s):
Therapeutics > Cardiovascular
2019-600 TREATMENT FOR RESTORING UREAGENESIS IN CARBAMOYL PHOSPHATE SYNTHETASE 1 DEFICIENCY
Treatment for Restoring Ureagenesis in Carbamoyl Phosphate Synthetase 1 DeficiencyTech ID: 30356 / UC Case 2019-600-0SUMMARYUCLA researchers in the Department of Surgery have developed a gene therapy to treat carbamoyl phosphate synthetase 1 deficiency.BACKGROUNDCarbamoyl phosphate synthetase 1 (CPS1) deficiency is an inherited autosomal recessive urea...
Published: 2/14/2025
|
Inventor(s):
Gerald Lipshutz
Keywords(s):
Cns
,
Gene Editing Systems
,
Gene Therapy
,
Genetic Disease
,
Therapeutics & Vaccines
Category(s):
Therapeutics
,
Therapeutics > CNS and Neurology
,
Therapeutics > Gene Therapy And Editing
2019-550: A Method to Prevent the Myelin Abnormalities Associated with Arginase Deficiency
SUMMARY UCLA researchers in the Department of Surgery have developed a gene therapy to prevent dysmyelination (and other CNS abnormalities) as a result of arginase deficiency. BACKGROUND Arginase deficiency (ARG1 deficiency, argininemia) is an inherited autosomal recessive disorder caused by deficient/defective enzyme arginase 1 in the final...
Published: 2/14/2025
|
Inventor(s):
Gerald Lipshutz
Keywords(s):
Cns
,
Gene Editing Systems
,
Gene Therapy
,
Genetic Disease
,
Therapeutics & Vaccines
Category(s):
Therapeutics
,
Therapeutics > CNS and Neurology
,
Therapeutics > Gene Therapy And Editing
2009-533 A MOLECULAR PATHWAY FOR IMPORTING RNA INTO MITOCHONDRIA
Case No. 2009-533SUMMARYResearchers at UCLA’s Jonsson Comprehensive Cancer Center, the Department of Chemistry and Biochemistry, and the Department of Pathology and Laboratory Medicine have uncovered a role for an essential cell protein, polynucleotide phosphorylase (PNPASE) in shuttling RNA into the mitochondria, the energy-producing “power...
Published: 2/14/2025
|
Inventor(s):
Michael Teitell
,
Carla Koehler
,
Geng Wang
Keywords(s):
Genetic Disease
,
Oncology
,
Therapeutics & Vaccines
Category(s):
Therapeutics
,
Therapeutics > Oncology
2017-701 Nanoparticle-Enabled Delivery of Large Plasmid DNA for Gene Therapy of Duchenne Muscular Dystrophy and Cancer
Polyrotaxane Nanoparticles for Delivery of Large Plasmid DNA in Duchenne Muscular DystrophySUMMARYUCLA researchers have designed, synthesized, and validated a polyrotaxane nanocarrier for targeted delivery of large plasmids for gene therapy applications for treatment of Duchenne muscular dystrophy and cancer.BACKGROUNDRapid development of genome/epigenome...
Published: 2/14/2025
|
Inventor(s):
Melissa Spencer
,
April Pyle
,
Huan Meng
,
Courtney Young
,
Xiangsheng Liu
Keywords(s):
Drug Delivery
,
Gene Editing Systems
,
Gene Therapy
,
Genetic Disease
,
Life Science Research Tools
,
Medical Devices and Materials
,
Musculoskeletal Disease
,
Nanoparticles
,
Oncology
,
PEG
,
Therapeutics & Vaccines
Category(s):
Life Science Research Tools
,
Medical Devices
,
Platforms > Drug Delivery
,
Therapeutics
,
Therapeutics > Gene Therapy And Editing
,
Therapeutics > Musculoskeletal Disease
,
Platforms > Drug Delivery > Nanoparticles
,
Therapeutics > Oncology
,
Platforms > Drug Delivery > PEG
1
2