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SYNGAP1 splice-switching antisense oligonucleotide as therapy for human cognitive disorders

Unmet Need: SYNGAP1-related Intellectual Disability (SRID, MRD5) is a severe neurodevelopmental disorder (NDD) characterized by encephalopathy, intellectual disability (ID), autism spectrum disorder (ASD), and epilepsy and accounts for 0.5-1% of all NDDs and ~1% of the ~200 million ID cases worldwide (SYNGAP Research Fund, 2020). Supportive management...

Published: 5/10/2024 | Inventor(s): Richard Huganir, Yoichi Araki, Ingie Hong, Richard Johnson, Yinuo Han

Keywords(s):

Category(s): Technology Classifications > Research Tools > Vectors & Plasmids

Compositions of Kinetic Nanoparticles containing Nucleic Acids, Polycations, and Lipids with Defined Sizes, and Methods of Producing the Same

Value Proposition:· Scalable, efficient, and tunable nanoparticle creation technique that can deliver a variety of payloads· Polymer-lipid nanoparticles are biodegradable and are being tested for immunogenicity· Can be used for in vivo gene therapy delivery as well as in vitro cell transfection and research applications Technology...

Published: 5/10/2024 | Inventor(s): Hai-Quan Mao, Stephany Tzeng, Jordan Green, Yizong Hu, Sashank Reddy, Leonardo Cheng

Keywords(s):

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

Targeted capture of custom nucleotide sequences for single-cell sequencing

Value Proposition· Implementation only requires simple modification of current capture beads.· Allows for targeting of custom sequences of interest that may be cell-type specific or expressed at low levels.· Targets RNA molecules without polyA tails.· Inexpensive modification to existing commercial methods. Technology DescriptionResearchers...

Published: 5/14/2024 | Inventor(s): Jonathan Ling, Katherine Irwin, Irika Sinha, Philip Wong, Linda Orzolek, Tyler Creamer

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Category(s): Technology Classifications > Research Tools > Nucleic Acids, Technology Classifications > Research Tools > Vectors & Plasmids, Technology Classifications > Research Tools > Assays

Induced Pluripotent Stem Cell Lines (BC1 and C7) and Plasmids (pEB-C5, pEB-Tg)

The Material consists of two induced pluripotent stem (iPS) cell lines derived from human bone marrow and cord blood.and plasmids used to create these virus-free and integration-free human iPS cell lines.

Published: 5/9/2024 | Inventor(s): Linzhao Cheng

Keywords(s): Basic Research Biomarker, Cell Model, Cloning Reagents, Discovery/Research Tools, In Vitro Research Tool, Plasmid, Research Reagent, Stem Cells

Category(s): Technology Classifications > Research Tools > Cell Lines, Technology Classifications > Research Tools > Stem Cells, Technology Classifications > Research Tools > Vectors & Plasmids, Technology Classifications > Research Tools

Single Vector Genome Editing with CRISPR/Cas and BacMam

Unmet NeedTargeted, site-specific genome editing is an important scientific tool with broad applications in basic science, the pharmaceutical industry, and translational medicine. It enables the addition of florescent or other types of gene tags for purification, visualization, or tracking. Genome editing can also introduce novel functions for endogenous...

Published: 5/9/2024 | Inventor(s): Chulan Kwon, Renjun Zhu, Amir Saberi

Keywords(s): Basic Research Biomarker, Cloning Reagents, Discovery/Research Tools, Other Virus, Plasmid, Research Reagent, Translational Research Biomarker, Viruses

Category(s): Technology Classifications > Research Tools > Vectors & Plasmids, Technology Classifications > Research Tools > Viruses

Compositionally Defined Plasmid DNA/Polycation Nanoparticles and Methods for Making the Same

Unmet NeedGene therapy can be a cure-all for genetic disorders, and can be a promising therapy for diseases such as cancer, neurodegenerative diseases, neurological disorders, heart disease or diabetes. The success of gene therapy is dependent on the safety, efficacy, and specificity of the treatment. The delivery vehicle, also known as a vector, is...

Published: 5/9/2024 | Inventor(s): Hai-Quan Mao, Yizong Hu, Martin Pomper, Heng-wen Liu, Il Minn, Christopher Ullman, Christine Carrington

Keywords(s): Drug Delivery Vehicle, Drug Delivery Vehicle Synthesis Method, Nanoparticles, Therapeutic Matter, Therapeutics

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

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

Scalable Method for Multiplexed Genome Editing

Unmet NeedTargeted genome editing has become a very important genetic tool with broad applications across various areas of science including but not limited to basic science, the pharmaceutical industry, and translational medicine. Presently, one of the most commonly used site-specific, genome editing systems is CRISPR/Cas9, as it enables insertion...

Published: 5/9/2024 | Inventor(s): Chulan Kwon, Renjun Zhu, Amir Saberi

Keywords(s): Adeno-Associated Virus, Basic Research Biomarker, Cell Lines, Discovery/Research Tools, In Vitro Research Tool, Research Reagent, Viruses

Category(s): Technology Classifications > Research Tools > Cell Lines, Technology Classifications > Research Tools > Vectors & Plasmids

Plasmid Expressing P. Falciparum Aldolase Isolate 3D7

Unmet NeedApproximately 214 million malaria cases were recorded in 2015 with 438,000 people dying from this disease. The majority of these deaths result from a failure to diagnose and treat malaria before the onset of severe disease, at which point, even with proper treatment a 20% mortality rate may be expected. Microscopy is the current gold standard...

Published: 5/9/2024 | Inventor(s): David Sullivan

Keywords(s): Basic Research Biomarker, Cloning Reagents, Discovery/Research Tools, Plasmid, Research Reagent

Category(s): Technology Classifications > Research Tools > Vectors & Plasmids, Technology Classifications > Research Tools

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