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.

This technology utilizes controlled-release microparticle compositions encapsulating CCL22 chemotactic factor to recruit regulatory T cells, thereby modulating immune responses and enhancing repair in acute skeletal muscle injuries.

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.

University of Pittsburgh researchers have designed innovative nucleotide prodrugs that leverage boryl allyl phosphate groups to selectively target oxidative stress environments.

This invention comprises recombinant adeno-associated viral (AAV) vectors engineered to simultaneously deliver and overexpress key mitochondrial import complex proteins (TIM17, TIM23, and TIM50) directly to neurons.

This technology comprises a novel class of "potentiator polypeptides", proteins co-administered with adeno-associated viruses (AAVs), that dramatically enhance viral tissue penetration, spread and infectivity by precisely and temporarily reducing the structural and molecular barriers that normally trap AAVs near the injection site.

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).

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

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%.

This technology introduces novel antisense oligonucleotide inhibitors targeting MYC-bound enhancer RNA, specifically GREB1 (MERG1), to effectively suppress cancer cell proliferation across multiple malignancies by modulating gene expression pathways critical for oncogenesis.

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.