Cell & Gene Therapy

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.

The University of Pittsburgh has developed a technology that enhances in vivo vasculogenic reprogramming using Tissue Nanotransfection (TNT) to deliver specific transcription factors.

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.

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.

University of Pittsburgh researchers have developed an innovative approach to enhance the effectiveness of adoptive cell therapy for cancer treatment by redirecting glucose metabolism.

This invention provides methods for preventing and treating hypertrophic cardiomyopathy by inhibiting key protein interactions (PCNA and POLD1).

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.

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.

Cancers of the blood account for an estimated 8% of all cancers in adults, with 5-year survival projections as low as 47%.

A University of Pittsburgh researcher is developing gene therapy for Prader-Willi syndrome (PWS).

University of Pittsburgh researchers have developed a viral vector that allows for the overexpression of Cdkn1a cDNA selectively in cardiomyocytes.

These human antibodies target the protein, protogenin, to reduce growth of medulloblastomas.

University of Pittsburgh and University of Colorado Boulder researchers have developed regulatory T cell therapy to treat reperfusion injury following myocardial infarction (MI, i.

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.

This approach employs targeted reduction of β-catenin expression or activity in the liver to alleviate porphyria by correcting dysregulated heme biosynthesis.