Available Technologies

Browse Penn-owned technologies available for licensing.

Search Results - gene+therapy

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Amniotic Fluid Stabilized Lipid Nanoparticles (LNPs) for In Utero Intra-Amniotic mRNA Delivery

Nanoparticles for potent encapsulation and delivery of therapeutic molecules for prenatal fetal therapy in the amniotic sac Problem: Many prenatal genetic diseases suffer from ineffective postnatal treatments. For this reason, therapies that are delivered to the fetus before birth hold promise to treat diseases prior to or in the early stages of...

Published: 10/11/2022

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Versatile virtual reality system for functional vision evaluation

​​Virtual reality-based system and software for quick, accurate, and reproducible evaluation of functional vision capacity following clinical treatment Problem: In 2013, the total economic burden of eye disorders and vision loss in the United States was $139 billion with projections to increase by ~20% over the following 10 years. Developing treatments...

Published: 12/10/2021

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Efficient and controllable genome editing complex

A split enzyme complex enabling precise gene editing Technology Overview: The ability to target DNA deaminases to specific loci in concert with CRISPR/Cas systems has opened up new frontiers with the potential to transform biology and medicine by allowing for precise genome editing. At the same time, current DNA “base editors” typically...

Published: 6/6/2022

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High performance CRISPR-Cas12a system for combinatorial genetic manipulation

CRISPR-Cas12a allowing for simultaneous editing of multiple genes in mammalian cells Problem: CRISPR-based gene editing technique has revolutionized the field of molecular biology. While CRISPR-Cas9-based methods are effectively used for single-gene knockout, multiplex gene editing remains a major challenge. Unlike Cas9, Cas12a can be used for multigene...

Published: 9/13/2022

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Gene therapy for treatment of Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy (DMD) therapy deletes let-7c miRNA binding sites using CRISPR-Cas9 based genome editing and AAV based delivery. Problem: Duchenne Muscular Dystrophy (DMD) is a fatal disease characterized by progressive muscle degeneration and weakness that affects 1 in 3,500 live-born males worldwide, and which ultimately causes death...

Published: 8/22/2022

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Gene Therapy for Macular Degeneration

Gene therapy targeting disposal of toxic intracellular lipid debris and lipofuscin in wet age related macular degeneration. Problem: Macular degeneration is a genetic eye disorder that affects the retina resulting in progressive vision loss. Macular degeneration has two main forms, Stargardt’s macular degeneration (STGD) which affects juveniles,...

Published: 8/22/2022

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Photolinker to cage biological molecules

Turn Genes On or Off with Light Technology Overview: Dmochowski lab has designed a Ruthenium-based compound, RuBEP, that is a synthetically versatile photolinker. The photolinker can be tuned to be activated at a large range of visible and near-IR wavelengths, allowing for better tissue penetration and reducing toxicity issues. The photoactivation...

Published: 9/13/2022

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One-step synthesis of cationic steroids for gene delivery, anti-inflammatory activity, and antimicrobial activity.

Reagents for improved DNA lipofection, depot anti-inflammation, and/or topical antimicrobial therapy Technology Overview: Researchers in the Diamond Lab have developed a versatile new class of pharmacologically-active molecules with multiple applications. These molecules deliver plasmids (pDNA), adenovirus (AdV), and adeno-associated virus (AAV)...

Published: 10/13/2022

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Gene therapy for the treatment of retinal degeneration and vision loss

Recombinant adeno-associated virus (rAAV) expressing the gene for retinitis pigmentosa GTPAse regulation (RPGR) for the functional and structural rescue of photoreceptor cells of the eye as gene therapy for retinal degeneration and vision loss. Technology Overview: Retinitis pigmentosa (RP) is a genetic form of early-onset retinal degeneration that...

Published: 10/30/2021

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