UCLA Researchers & Innovators
Industry & Investors
News & Events
About
Concierge
Search Results - genetic+disease
17
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: 9/15/2023
|
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: 1/23/2024
|
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
2019-772: A Single Chain Antibody That Binds Tau Oligomers and Inhibits Seeding by Pathological Extracts From Alzheimer’s Disease
SUMMARYUCLA researchers in the Department of Chemistry & Biochemistry have developed a method for efficiently producing tau oligomers and a single-chain antibody against oligomeric tau protein with potential for theranostic utility in the treatment of Alzheimer’s and other tauopathies.BACKGROUND:Alzheimer’s disease (AD) is the most prevalent...
Published: 9/5/2023
|
Inventor(s):
David Eisenberg
Keywords(s):
Amyloid
,
Antibodies
,
Central Nervous System
,
Genetic Disease
,
Life Science Research Tools
,
Neurodegeneration Alzheimer's Disease Predictive Testing
,
Tau Protein
Category(s):
Life Science Research Tools > Antibodies
,
Therapeutics > CNS and Neurology
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: 9/20/2023
|
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: 9/20/2023
|
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: 7/19/2023
|
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: 7/19/2023
|
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: 9/5/2023
|
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: 9/5/2023
|
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: 7/19/2023
|
Inventor(s):
Michael Teitell
,
Carla Koehler
,
Geng Wang
Keywords(s):
Genetic Disease
,
Oncology
,
Therapeutics & Vaccines
Category(s):
Therapeutics
,
Therapeutics > Oncology
1
2