JOINT STOCK COMPANY "BIOCAD" (20240350663). ISOLATED MODIFIED AAV9 CAPSID PROTEIN VP1 simplified abstract

ISOLATED MODIFIED AAV9 CAPSID PROTEIN VP1

Organization Name

JOINT STOCK COMPANY "BIOCAD"

Inventor(s)

Anna Nikolaevna Strelkova of Yaransk (RU)

Tatiana Evgenievna Shugaeva of Moscow (RU)

Pavel Mikhailovich Gershovich of Saint Petersburg (RU)

Pavel Andreevich Iakovlev of Saint Petersburg (RU)

Dmitry Valentinovich Morozov of Saint-Petersburg (RU)

ISOLATED MODIFIED AAV9 CAPSID PROTEIN VP1 - A simplified explanation of the abstract

This abstract first appeared for US patent application 20240350663 titled 'ISOLATED MODIFIED AAV9 CAPSID PROTEIN VP1

The present application pertains to gene therapy and molecular biology, specifically focusing on an isolated modified capsid protein VP1 from adeno-associated virus serotype 9 (AAV9).

• The invention involves one or more amino acid substitutions in the VP1 protein of AAV9 compared to the wild-type, enhancing the efficiency of vector production based on recombinant AAV9.
• The modified capsid protein VP1 leads to improved assembly of the vector, which is crucial for gene therapy applications.
• The innovation extends to the development of a capsid and a vector based on the modified VP1 protein, offering new possibilities for gene delivery systems.
• The technology opens up opportunities for enhanced gene therapy treatments by optimizing the production process of the vector.

Potential Applications: - Gene therapy for various genetic disorders - Targeted delivery of therapeutic genes - Research in molecular biology and genetic engineering

Problems Solved: - Enhancing the efficiency of vector production - Improving the effectiveness of gene therapy treatments

Benefits: - Increased success rates in gene therapy applications - Enhanced precision in gene delivery - Potential for developing new therapeutic interventions

Commercial Applications: Title: Enhanced Vector Production Technology for Gene Therapy Applications This technology could revolutionize the gene therapy industry by streamlining vector production processes, leading to more effective treatments and potentially opening up new markets for gene therapy products.

Prior Art: Readers interested in exploring prior art related to this technology could start by researching advancements in AAV vector technology and modifications to capsid proteins in gene therapy applications.

Frequently Updated Research: Stay updated on the latest research in AAV vector technology, capsid protein modifications, and gene therapy advancements to leverage the full potential of this innovative technology.

Questions about Gene Therapy: 1. How does the modified capsid protein VP1 improve the efficiency of vector production in gene therapy applications? - The modified VP1 protein enhances the assembly of the vector, leading to more effective gene delivery and potentially improving treatment outcomes.

2. What are the potential implications of using recombinant AAV9 vectors based on the modified VP1 protein in gene therapy research? - The use of these vectors could significantly advance gene therapy research by offering a more efficient and precise delivery system for therapeutic genes.

Original Abstract Submitted

the present application relates to the fields of gene therapy and molecular biology. more specifically, the present invention relates to an isolated modified capsid protein vp1 from adeno-associated virus serotype (aav9) comprising one or more amino acid substitutions compared to the wild-type aav9 capsid protein vp1, which substitutions increase the efficiency of production (assembly) of the vector based on recombinant adeno-associated virus serotype 9 (raav9), to a capsid and a vector based on the above vp1, as well as to uses thereof.