Search Results - nucleic+acid

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SYNGAP antisense oligonucleotide as therapy for human cognitive disorders

Unmet Need: Recent human genetic studies have suggested that mutations in the SYNGAP1 gene are linked to intellectual disability (ID), autism spectrum disorders (ASD), neurodevelopmental disorders, high rates of epilepsy, and schizophrenia. SYNGAP1 is a gene that encodes SynGAP, a GTPase-activating protein that is highly enriched in nerve cells in the...

Published: 5/9/2024 | Inventor(s): Richard Huganir, Ingie Hong, Yoichi Araki

Keywords(s): Biologics, CNS and Neurological Disorders, Cognitive Impairment, Disease Indication, Gene Therapy, Nucleic Acid, Single, Target, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations > Psychiatry > Autism Spectrum Disorder, Clinical and Disease Specializations > Psychiatry > ADHD, Clinical and Disease Specializations > Psychiatry > Mood Disorders, Technology Classifications > Therapeutic Modalities > Gene Therapies

Bioreducible Poly(beta-amino ester)s for siRNA Delivery

C12218: Efficient Delivery Vehicle for Controlled Gene Knockdown Novelty: This technology comprises the use of bioreducible polymer based nanoparticle system that can release siRNA cargo to the cytoplasm of cells to knockdown target gene expression in tissues. Value Proposition: Gene therapy is a developing medical field and there is a need for...

Published: 5/9/2024 | Inventor(s): Kristen Kozielski, Stephany Tzeng, Jordan Green

Keywords(s): Biologics, Brain Cancer, Cancers, Cell Therapy, Disease Indication, Drug Delivery Vehicle, Glioma, Nucleic Acid, Polymers, Single, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Neurology, Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Cell Therapies, Technology Classifications > Therapeutic Modalities > Dendrimers & Polymers, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Oncology > Brain Cancer, Clinical and Disease Specializations > Oncology > Glioma

Antisense oligonucleotide therapy and biomarker assay for C9ORF72 hexanucleotide expansion disorders

Therapy to Treat and Assay to Monitor Neurodegenerative Disease JHU REF: [C12195]Invention novelty: This technology is the use of antisense oligonucleotides (ASO) as a therapeutic for specific neurodegenerative diseases. The technology could also be used in development of a biomarker assay to monitor the efficacy of the ASO therapy. Value Proposition: Current...

Published: 5/9/2024 | Inventor(s): Christopher Donnelly, C. Bennett, Susan Freier, Rita Sattler, Jeffrey Rothstein

Keywords(s): Amyotrophic Lateral Sclerosis, Assay, Biologics, Clinical Diagnostics, CNS and Neurological Disorders, Dementia, Disease Indication, In Vitro Diagnostics, Neurodegeneration, Nucleic Acid, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Clinical and Disease Specializations, Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Diagnostics > In Vitro Diagnostics, Technology Classifications > Research Tools > Assays, Technology Classifications > Diagnostics, Clinical and Disease Specializations > Neurology, Technology Classifications > Research Tools, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Dementia, Clinical and Disease Specializations > Neurology > Neurodegeneration

Use of Combination Retinoic Acid and dsRNA Products for Skin Rejuvenation

Unmet NeedGlobally, the anti-aging cosmetic treatment market is projected to grow to $57.5B dollars in 2020. Currently, one of the leading the leading topical ingredients in anti-aging creams for wrinkle reduction is retinoids, which are derivatives of vitamin A. Retinoic acid, the active metabolite of retinoids, has been shown to help repair sun damaged...

Published: 5/9/2024 | Inventor(s): Luis Garza, Dongwon Kim

Keywords(s): Biologics, Chemotherapy/Immunotherapy, Combination, Disease Indication, Nucleic Acid, Skin Disorders, Small Molecules, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type, Wound Healing

Category(s): Technology Classifications > Therapeutic Modalities

A Membrane-targeting Construct of NF1 GAP-related Domain (GRD) that Suppresses RAS Activity in NF1-related MPNST, NF1 and other Rasopathies

Unmet NeedNeurofibromatosis type 1 (NF1) is a common genetic disorder that affects approximately 1 in 3,000 individuals worldwide. Patients with NF1 have DNA alterations to the neurofibromin 1 gene which cause RASopathies, a family of diseases caused by altered Ras signaling. RASopathies can include both benign and malignant tumors, including malignant...

Published: 5/9/2024 | Inventor(s): Verena Staedtke, Renyuan Bai

Keywords(s): Adeno-Associated Virus, Biologics, Cancers, Discovery/Research Tools, Disease Indication, Nucleic Acid, Research Reagent, Target, Therapeutic Matter, Therapeutic Substance, Therapeutics, Translational Research Biomarker, Viruses

Category(s): Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Research Tools > Vectors & Plasmids

Transcriptionally Targeted and CPG-free Plasmid for Theranostic Gene Therapy

Unmet NeedHepatocellular carcinoma (HCC) is the third leading cancer in the US with a ~twenty percent (20%) survival rate. HCC develops typically in the inflammatory environment of the liver caused by infections or liver disease. Therefore, it is essential to have highly targeted anti-cancer therapies to achieve clinical efficacy. Currently, viral gene...

Published: 5/9/2024 | Inventor(s): Jordan Green, Martin Pomper, Camila Gadens Zamboni, Hannah Vaughan, Il Minn

Keywords(s): Biologics, Cancers, Disease Indication, Gene Therapy, Hepatocellular Carcinoma, Liver Cancer, Nucleic Acid, Single, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Biologics, Clinical and Disease Specializations > Oncology > Hepatocellular Carcinoma

A Novel Method to Efficiently Synthesize a Biodegradable Dextran to Use as an Efficient Cationic Nanopolymer Carrier for siRNA Delivery

Unmet NeedIn theory, RNA interference (RNAi) can be used to decrease expression of any gene driving a disease or pathology, but has seen limited clinical usage as gene therapy due to technical issues, i.e., small interfering RNA (siRNA) cannot enter cells efficiently due to their weight and charge. As such, a carrier molecule is needed to bring siRNA...

Published: 5/9/2024 | Inventor(s): Zaver Bhujwalla, Zhihang Chen

Keywords(s): Biologics, Cancers, Disease Indication, Drug Delivery Vehicle, Nucleic Acid, Polymers, Therapeutic Matter, Therapeutic Method, Therapeutic Substance, Therapeutics

Category(s): Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Therapeutic Modalities > Dendrimers & Polymers, Technology Classifications > Research Tools > Nucleic Acids

MicroRNA Delivery as a Novel Therapeutic Strategy for Liver Cancer

Unmet needOver 500,000 people are diagnosed with liver cancer worldwide each year. According the American Cancer Society, liver cancer resists most chemotherapy drugs and the most effective agents shrink less than 1 in 5 tumors and generally for only a short time. Hepatocellular carcinoma (HCC) is often diagnosed at an advanced stage making it difficult...

Published: 5/9/2024 | Inventor(s): Kathryn Mendell, Raghu Chivukula, Janaiah Kota, Erik Wentzel, Jerry Mendell, K. Clark, Joshua Mendell

Keywords(s): Biologics, Cancers, Disease Indication, Gene Therapy, Liver Cancer, Nucleic Acid, Single, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Hepatology, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Biologics, Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Oncology > Liver Cancer

Formulation Method and Use of Highly Stable Biodegradable Gene Vector Platforms Capable of Overcoming Various Biological Barriers

JHU researchers have formulated compact, colloidally stable gene carriers that have a dense surface coverage of hydrophilic and neutrally charged PEG. These gene vectors stably retain their physicochemical characteristics over at least a week in aqueous solution and are highly stable in physiological solutions. Moreover, they demonstrate the ability...

Published: 5/9/2024 | Inventor(s): Panagiotis Mastorakos, Jung Soo Suk, Justin Hanes

Keywords(s): Biologics, Drug Delivery Vehicle, Gene Therapy, Nucleic Acid, Plasmid/Vector, Single, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms

Modulation of Bio-electrical Rhythms Via a Novel Engineering Approach

Spontaneous cellular electrical rhythms govern numerous biological processes from the autonomous beating of the heart, to respiratory rhythms and insulin secretion. For instance, abnormal pacing in the heart leads to various forms of electrical disorders that necessitate conventional pharmacologic interventions and implantation of costly electronic...

Published: 5/9/2024 | Inventor(s): Heecheol Cho, Tian Xue, Eduardo Marban, Suk-Ying Tsang, Ronald Li

Keywords(s): Biologics, Gene Therapy, Nucleic Acid, Single, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities

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