University of Pittsburgh
Cell & Gene Therapy - University of Pittsburgh

Cell & Gene Therapy

Includes technologies from the cell and gene therapy space, including NK cells, stem cells, T cells, AAV, Adenovirus, CRISPR, DNA, Lentivirus, RNA and vaccinia.
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Bait Oligonucleotides to Prevent TDP-43 Associated Neurodegeneration
This invention is a novel class of short RNA oligonucleotides, known as RNA chaperones, designed to specifically engage and stabilize aggregation-prone RNA-binding proteins implicated in neurological disorders.
Therapeutic Modality
Gene Therapy - RNA
Neuroscience
Parkinson's DiseaseAmyotrophic lateral sclerosis (ALS)Alzheimer's Disease
Christopher Donnelly
Med-Neurobiology
2024-11-30
Vasculogenic Reprogramming for Diabetic Wound Healing and Ischemic Tissue Regeneration
The University of Pittsburgh has developed a technology that enhances in vivo vasculogenic reprogramming using Tissue Nanotransfection (TNT) to deliver specific transcription factors.
Therapeutic Modality
Gene Therapy - DNA
Endocrinology and Metabolic Diseases
Diabetes
Chandan Sen
Med-Surgery
2024-09-16
Novel Research Tool to Study TDP-43 Dysfunction
University of Pittsburgh researchers have developed a novel RNA biosensor named CUTS-1 (CFTR-UNC13A TDP-43 Loss of Function Sensor), capable of detecting TDP-43 dysfunction in cells.
Therapeutic Modality
Gene Therapy - RNA
Neuroscience
Alzheimer's Disease
Christopher Donnelly
Med-Neurobiology
2024-02-08
Restoring Sensation: Cutting-Edge Skin Reprogramming to Combat Diabetic Nerve Damage
This invention utilizes a unique cocktail of transcription factors, known as ASK, delivered via Tissue Nanotransfection (TNT) to non-invasively reprogram skin cells in vivo.
Therapeutic Modality
Gene Therapy - DNA
Neuroscience
Genetics
Chandan Sen
Med-Surgery
2024-01-29
A Novel Method to Improve Adoptive Cell Therapy
University of Pittsburgh researchers have developed an innovative approach to enhance the effectiveness of adoptive cell therapy for cancer treatment by redirecting glucose metabolism.
Therapeutic Modality
Cell Therapy - T Cell
Oncology
Greg Delgoffe
Med-Immunology
2020-07-16
Targeting Cardiomyocyte Endoreplication to Prevent Hypertrophic Cardiomyopathy
This invention provides methods for preventing and treating hypertrophic cardiomyopathy by inhibiting key protein interactions (PCNA and POLD1).
Therapeutic Modality
Gene Therapy - RNA
Cardiovascular
Hypertrophic cardiomyopathy
4
GeneticsRegenerative medicinePlatform Technology
Jason Becker
Med-Medicine
2025-03-30
Stimulation of Angiogenesis with miRNA as Protection Against Acute Kidney Injury
Hospital-acquired acute kidney injury (AKI) accounts for 22% of all cases worldwide, and an estimated 50% of critically ill inpatients are estimated to suffer from AKI.
Therapeutic Modality
Gene Therapy - RNA
Nephrology
Regenerative medicine
Sunder Lucas
Med-Pediatrics
2020-07-16
Producing Tumor Infiltrating T Cells for Immunotherapy
University of Pittsburgh researchers have developed a novel approach for isolating and expanding tumor-infiltrating γδ T cells (TILs) from solid tumors, presenting a significant advancement in immunotherapy.
Therapeutic Modality
Cell Therapy - T Cell
Oncology
Immuno-oncology
Michael Lotze
Med-Surgery
2019-12-07
Driving Oxidative Metabolism in Therapeutic T Cells through Overexpression of AMPK
Cancers of the blood account for an estimated 8% of all cancers in adults, with 5-year survival projections as low as 47%.
Therapeutic Modality
Gene Therapy - Lentivirus
Oncology
5
Chronic Myeloid Leukemia (CML)Chronic Lymphocytic Leukemia (CLL)Acute Myeloid Leukemia (AML)
Craig Byersdorfer
Med-Pediatrics
2020-08-26
Gene Therapy for Prader-Willi Syndrome
A University of Pittsburgh researcher is developing gene therapy for Prader-Willi syndrome (PWS).
Therapeutic Modality
Gene Therapy - AAV
Endocrinology and Metabolic Diseases
Prader-Willi Syndrome
Platform Technology
Robert Nicholls
Med-Pediatrics
2022-12-06
Innovative Gene Therapy for Heart Disease: p21 Overexpression in Cardiomyocytes
University of Pittsburgh researchers have developed a viral vector that allows for the overexpression of Cdkn1a cDNA selectively in cardiomyocytes.
Therapeutic Modality
Gene Therapy - AAV
Cardiovascular
Hypertrophic cardiomyopathy
Jason Becker
Med-Medicine
2024-04-03
Human Antibodies Identified as Brain Cancer Immunotherapy Targets
These human antibodies target the protein, protogenin, to reduce growth of medulloblastomas.
Therapeutic Modality
Cell Therapy - T Cell
Oncology
4
Diffuse intrinsic pontine gliomas (DIPG)MedulloblastomaBrain cancer - Other
PediatricsRare disease
Dimiter Dimitrov
2021-05-25
iHeart: Injectable Hydrogel for Enhancing or Attracting Regulatory T-Cells
University of Pittsburgh and University of Colorado Boulder researchers have developed regulatory T cell therapy to treat reperfusion injury following myocardial infarction (MI, i.
Therapeutic Modality
Cell Therapy - T Cell
Cardiovascular
Myocardial infarction
Matthew Borrelli
Chem/Petroleum Engineering
2023-08-15
Using Novel siRNAs to Treat Liver Failure
University of Pittsburgh researchers have identified key transcription factors involved in alcoholic hepatitis (AH) and developed a novel small interfering RNA (siRNA) to inhibit gene expression and improve hepatocyte function.
Therapeutic Modality
Gene Therapy - RNA
Hepatology
Ramon Bataller Alberola
2018-06-11
Beta-Catenin Inhibition Therapy for Treating Porphyria
This approach employs targeted reduction of β-catenin expression or activity in the liver to alleviate porphyria by correcting dysregulated heme biosynthesis.
Therapeutic Modality
Gene Therapy - RNA
Hematology
Rare disease
Satdarshan Monga
Med-Pharmacology and Chemical Biology
2018-02-15