A method for restoring BMP-receptor signaling in a cell

Simple SummaryContent extracted from patent full text and abstract with AI.

This patent describes a method for restoring or enhancing Bone Morphogenetic Protein (BMP)-receptor signaling in cells by increasing the activity of the protein cGKI (cGMP-dependent protein kinase type I). The invention demonstrates that boosting cGKI activity compensates for defects in the BMP signaling pathway, which is crucial in various physiological processes and diseases. Increased cGKI activity can be achieved using multiple approaches, such as overexpressing cGKI or its active forms, inhibiting its negative regulators, or providing BMP-receptor ligands. Additionally, cGKI can be used in drug screening and gene activation strategies related to BMP signaling.

Use CasesContent extracted from patent full text and abstract with AI.

• Treatment of pulmonary artery hypertension (PAH) by restoring BMP signaling in affected tissue.
• Development of new therapies for certain cancers that involve disrupted BMP-receptor pathways.
• Treatment for fibrotic diseases where BMP signaling is compromised.
• Therapeutic approaches for bone diseases, including slow- or non-healing fractures and bone malformations.
• Potential interventions for neurodegenerative diseases linked to BMP signaling defects.
• Drug screening to discover compounds that enhance cGKI activity for various diseases.
• Use in gene therapy or cellular engineering where activation of genes with BMP response elements is desired.

BenefitsContent extracted from patent full text and abstract with AI.

• Restores a key cellular signaling pathway implicated in multiple serious diseases.
• Provides a versatile toolkit: multiple methods to increase cGKI activity (protein delivery, gene transfection, inhibition of regulatory proteins, or use of constitutively active forms).
• Potentially overcomes limitations of therapies relying on large or mutated membrane receptors by targeting a smaller intracellular protein (cGKI).
• Enables pharmaceutical compositions targeting a range of diseases, broadening therapeutic applications.
• Allows for high-throughput screening of new drugs that modulate the BMP/cGKI pathway.
• Contributes to understanding and manipulation of gene expression where BMP response elements are involved, aiding regenerative and gene therapies.
• Can complement existing treatments, such as PDE5 inhibitors already used for PAH, by revealing and targeting their underlying mechanisms more precisely.

Technical Classifications (CPCs)

Inventors & Applicants

Patent Abstract

The invention relates to a method for restoring BMP-receptor signaling in a cell. According to the invention, the activity of the protein cGKI is increased in a cell. Furthermore, the invention relates to the use of cGKI for the treatment of a disease selected from the group consisting of pulmonary artery hypertension (PAH), cancer, fibrosis, bone diseases, and neurodegenerative diseases, and the use of cGKI for manufacturing a pharmaceutical composition for the treatment of said diseases, the use of a BMP receptor for screening for compounds having cGKI activity, the use of cGKI for screening for receptors associated with it, and the use of cGKI for the transcriptional activation of genes containing a BMP response element.