Search Results - adeno-associated+virus

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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: 9/26/2024 | 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: 9/27/2024 | 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

C38

C04744: C38 Technical Details: JHU scientists have created a Cystic Fibrosis bronchial epithelial cell line that was isolated from a patient with genotype ?508/W1282X. Cells were immortalized with adeno12SV0 and later transfected with a cDNA from AAV carrying CFTR truncated for the first 119 amino acids.Looking for Partners: This cell line may be...

Published: 9/26/2024 | Inventor(s): Pamela Zeitlin, William Guggino, Terence Flotte

Keywords(s): Adeno-Associated Virus, Basic Research Biomarker, Cell Model, Cystic Fibrosis, Cystic Fibrosis (CF), Discovery/Research Tools, Disease Indication, Epithelial Cells, Genetic Disorders, In Vitro Research Tool, Research Reagent, Translational Research Biomarker, Viruses

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Genetic Diseases > Cystic Fibrosis, Clinical and Disease Specializations > Rare Diseases, Clinical and Disease Specializations > Respiratory Diseases, Technology Classifications > Research Tools > Cell Lines, Technology Classifications > Research Tools > Viruses, Clinical and Disease Specializations > Genetic Diseases, Technology Classifications > Research Tools