Search Results - gene+therapy

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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

Method for using alternative splicing to control specificity of gene therapy

Unmet NeedViral-based gene therapy holds tremendous promise for the treatment of human diseases. Using genetically engineered carriers called vectors to deliver genes into cells, gene therapy can help replace a mutated gene with a healthy copy or even introduce a new gene into the body to fight a disease. However, an important concern when designing...

Published: 5/9/2024 | Inventor(s): Jonathan Ling, Seth Blackshaw

Keywords(s): Drug Delivery Mechanism, Gene Therapy, Single, Therapeutic Matter, Therapeutics, Therapy Type

Category(s): Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Research Tools > Vectors & Plasmids

14-3-3 Mediates a p53-Induced G2 Arrest

C03338: 14-3-3 Mediates a p53-Induced G2 Arrest Technical Details: Exposure of colorectal cancer (CRC) cells to ionizing radiation results in a cell-cycle arrest in G1 and G2. The G1 arrest is due to p53-mediated induction of the cyclin-dependent kinase inhibitor p21WAF1/CIP1/SDI1, but the basis for the G2 arrest is unknown. Through a quantitative...

Published: 5/9/2024 | Inventor(s): Heiko Hermeking, Kenneth Kinzler, Bert Vogelstein

Keywords(s): Biomarker, Cancers, Cell Lines, Cell Signaling, Discovery/Research Tools, Disease Indication, Gene Therapy, Human Cell Lines, In Vitro Research Tool, Molecular Switches/Receptors, Pharmakodynamic Biomarker, Research Reagent, Single, Therapeutic Matter, Therapeutics, Therapy Type, Translational Research Biomarker

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Oncology, Technology Classifications > Research Tools > Nucleic Acids, Technology Classifications > Diagnostics > Biomarkers, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Research Tools > Proteins, Ligands & Receptors, Technology Classifications > Diagnostics, Technology Classifications > Research Tools, Technology Classifications > Therapeutic Modalities

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

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

Molecular Switches and Methods for Making and Using the Same Involving the Circular Permutation of DNA

Technical Details: The invention provides molecular switches which couple external signals to functionality and to methods of making and using the same. The switches according to the invention can be used, for example, to regulate gene transcription, target drug delivery to specific cells, transport drugs intracellularly, control drug release, provide...

Published: 5/9/2024 | Inventor(s): Gurkan Guntas, Marc Ostermeier

Keywords(s): Basic Research Biomarker, Cell Signaling, Discovery/Research Tools, Gene Therapy, Molecular Switches/Receptors, Research Reagent, Single, Therapeutic Matter, Therapeutic Substance Synthesis Method, Therapeutics, Therapy Type

Category(s): Technology Classifications > Research Tools > Proteins, Ligands & Receptors, Technology Classifications > Research Tools, Technology Classifications > Therapeutic Modalities, Technology Classifications > Therapeutic Modalities > Gene Therapies

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

scAAV.EF1a.miR26a.eGFP Plasmid

C10912: A plasmid to produce recombinant adeno-associated virus (AAV) that expresses microRNA-26a and green fluorescent proteinValue Proposition: • miR-26a, cloned into the short intron that is part of the EF1a promoter unit, thus allowing simultaneous production of eGFP and miR-26a from a single transcript • efficient expression of both...

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

Keywords(s): Cancers, Disease Indication, Drug Delivery Vehicle, Gene Therapy, Liver Cancer, Plasmid/Vector, Single, Therapeutic Matter, Therapeutics, Therapy Type

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

C03416: HIF-1aDP, A Constitutively-Expressed form of Hypoxia-Inducible Factor 1a

C03416: HIF-1aDP, A Constitutively-Expressed form of Hypoxia-Inducible Factor 1a Technical Details: It has previously been demonstrated that a truncation of HIF-1alpha after amino acid residue 390 results in a constitutively expressed form of the protein. The purpose of this work was to identify mechanisms by which the half-life of the HIF1alpha...

Published: 5/9/2024 | Inventor(s): Gregg Semenza

Keywords(s): Biologics, Cardiovascular Diseases, Disease Indication, Gene Therapy, Natural compounds, Non-novel, Predicted Novelty, Protein, Single, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Cardiovascular, Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Proteins, Technology Classifications > Therapeutic Modalities

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