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RNA-Aptamer-Mediated Base Editing System
​ ​ Invention Summary: The CRISPR/Cas9 system is a powerful tool for targeted genetic engineering in organisms ranging from animals to plants. Cas9 has intrinsic nuclease activity, which causes sequence specific DNA double-strand breaks (DSBs), leading to activation of cellular pathways such as homology dependent repair (HDR) for gene conversion...
Published: 5/10/2022   |   Inventor(s): Shengkan (Victor) Jin, Juan-Carlos Collantes
Keywords(s):  
Category(s): Technology Classifications > Healthcare & Life Sciences, Technology Classifications > Therapeutics, Technology Classifications > Research Tools, Technology Classifications > Gene Therapy
MicroRNA-7 Gene Therapy Promotes Functional Recovery Following Spinal Cord Injury
Following sever spinal cord compression, the AAV-miR-7 injected mice showed improved locomotor recovery, which is characterized by the Basso Mouse Scale (BMS). Invention Summary: Spinal cord injury (SCI) results in partial or complete loss of sensory or motor function. According to the National Spinal Cord Injury Statistical Center,...
Published: 12/22/2021   |   Inventor(s): Mary Maral Mouradian, Eunsung Junn, Myung-Sik Yoo
Keywords(s): Spinal Cord
Category(s): Technology Classifications > Healthcare & Life Sciences, Technology Classifications > Therapeutics, Technology Classifications > Gene Therapy
A Chemogenetic Therapy for Neuropathic Pain
Invention Summary: Neuropathic pain (NP) typically arises as a consequence of a lesion or disease affecting peripheral nerve fibers. Prominent features in the pathophysiology of neuropathic pain include increased, ectopic neuronal activity in pain pathways. In general, NP is frequently refractory to conventional analgesics and currently there are...
Published: 10/5/2021   |   Inventor(s): Gary Aston-Jones
Keywords(s): Neurological disorder & neuropathic pain
Category(s): Technology Classifications > Healthcare & Life Sciences, Technology Classifications > Therapeutics, Technology Classifications > Gene Therapy
dCas9 Protein Allele Sequestration for Improved CRISPR Gene Editing
style="line-height:normal">​ Invention Summary: CRISPR technology is widely used in research to edit genes in animals and cell lines and is moving towards therapeutic use in the clinic. Inherent to almost all methods employed in gene editing is the lack of control in target gene usage. A method to direct cleavage of only a single target within...
Published: 3/22/2021   |   Inventor(s): Peter Romanienko, Ghassan Yehia
Keywords(s): Gene editing
Category(s): Technology Classifications > Healthcare & Life Sciences, Technology Classifications > Gene Therapy, Technology Classifications > Research Tools
Novel Gene Therapy to Treat L-DOPA-Induced Dyskinesia
Viral vector injections of rAAV-∆FosB shRNA significantly reduced abnormal involuntary movements (AIMs) compared to control vector in a rat model of Parkinson’s disease. Invention Summary: Parkinson's disease (PD) is a progressive, debilitating, and currently incurable neurodegenerative disease. Long-term dopamine replacement therapy in...
Published: 8/18/2020   |   Inventor(s): Mary Maral Mouradian, Stella Papa
Keywords(s): Neurological disorder & neuropathic pain
Category(s): Technology Classifications > Healthcare & Life Sciences, Technology Classifications > Gene Therapy, Technology Classifications > Therapeutics
Personalized and Timed Release of Biomolecules
Invention Summary: Protein/peptide-based therapeutics are powerful medications, however their use is often hampered by very short half-lives and the necessity for intravenous or subcutaneous injections. Researchers from Rutgers University and Massachusetts General Hospital have developed a novel method to deliver protein/peptide-based therapeutics...
Published: 3/2/2020   |   Inventor(s): Biju Parekkadan, Alexandra Burr, Alfred Tamayo
Keywords(s): Stem Cells
Category(s): Technology Classifications > Healthcare & Life Sciences, Technology Classifications > Physical Sciences & Engineering, Technology Classifications > Medical Devices, Technology Classifications > Cell Therapy, Technology Classifications > Gene Therapy
New Targeted Gene Therapy for Treatment of Duchenne Muscular Dystrophy
Invention Summary: Duchenne muscular dystrophy (DMD) is the most common and severe form of muscular dystrophy. It is a genetic disorder and patients suffering from DMD experience gradual muscle weakness in early childhood and are non-ambulant by 12 years of age. The progressive nature of this disease leads to restrictive pulmonary function, diaphragm...
Published: 6/13/2019   |   Inventor(s): Gopal Babu
Keywords(s): Neurological disorder & neuropathic pain
Category(s): Technology Classifications > Healthcare & Life Sciences, Technology Classifications > Gene Therapy, Technology Classifications > Therapeutics