Search Results - liudmila+cebotaru

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Cystic Fibrosis Transmembrane Conductance Regulator Modulators for Treating Autosomal Resessive Polycystic Kidney Disease

Unmet Need / Invention Novelty: Autosomal recessive polycystic kidney disease (ARPKD) patients that survive beyond neonatal age face various comorbidities that progress into chronic liver disease and end stage kidney disease. There is no cure for ARPKD and current treatments are limited to supportive treatments that manage symptoms and preserve kidney...

Published: 6/28/2024 | Inventor(s): Liudmila Cebotaru

Keywords(s):

Category(s): Clinical and Disease Specializations > Nephrology > Polycystic Kidney Disease, Technology Classifications > Therapeutic Modalities > Targets

Use of CFTR Modulators for the Treatment of ADPKD

Unmet Need: Autosomal dominant polycystic kidney disease (ADPKD) is characterized by the progressive enlargement of multiple renal cysts, leading to chronic pain, hypertension, and decline in renal function and renal failure in a substantial subset of patients. Fluid enters the cysts via a cAMP-dependent process involving the cystic fibrosis transmembrane...

Published: 6/27/2024 | Inventor(s): Liudmila Cebotaru

Keywords(s): Biologics, Protein, Therapeutic Matter, Therapeutic Substance, Therapeutics

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

ABCA4 Cell Lines

TITLE: ABC4A cell linesCASE NUMBER: C13886ABSTRACTStargardt disease is the most common form of early onset macular degeneration. Mutations in ABCA4, a member of the ATP-binding cassette (ABC) family, are associated with Stargardt disease. JHU researchers have examined two disease-causing mutations in the NBD1 region of ABCA4, R1108C and...

Published: 6/27/2024 | Inventor(s): Liudmila Cebotaru

Keywords(s): Cell Lines, Discovery/Research Tools, Disease Indication, Eye Disorders, Human Cell Lines, In Vitro Research Tool, Macular Degeneration, Neurodegeneration

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Neurology > Neurodegeneration, Clinical and Disease Specializations > Ophthalmology > Macular Degeneration, Technology Classifications > Research Tools > Cell Lines, Technology Classifications > Research Tools > Human Cell Lines, Clinical and Disease Specializations > Neurology, Clinical and Disease Specializations > Ophthalmology, Technology Classifications > Research Tools