Looking for breakthrough ideas for innovation challenges? Try Patsnap Eureka!

Materials and methods for treatment of pain related disorders

A gene and cell technology, applied in the field of alteration of sodium channel alpha subunit 9 gene

Pending Publication Date: 2019-06-04
VERTEX PHARMA INC
View PDF193 Cites 0 Cited by
  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

These newer platforms offer a greater degree of reproducibility, but still have their limitations

Method used

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
View more

Image

Smart Image Click on the blue labels to locate them in the text.
Viewing Examples
Smart Image
  • Materials and methods for treatment of pain related disorders
  • Materials and methods for treatment of pain related disorders
  • Materials and methods for treatment of pain related disorders

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0528] Example 1 - CRISPR / SpCas9 Target Sites of the SCN9A Gene

[0529] The region of the SCN9A gene was scanned for target loci. Each region was scanned for a protospacer adjacent motif (PAM) with sequence NRG. The gRNA 20bp spacer sequence corresponding to the PAM was then identified as shown in SEQ ID NO: 18989-56863 of the Sequence Listing.

Embodiment 2

[0530] CRISPR / SaCas9 target site of embodiment 2-SCN9A gene

[0531] The region of the SCN9A gene was scanned for target loci. Each region was scanned for a protospacer adjacent motif (PAM) with the sequence NNGRRT. The gRNA 20bp spacer sequence corresponding to the PAM was then identified as shown in SEQ ID NO: 8562-13614 of the Sequence Listing.

Embodiment 3

[0532] Example 3 - CRISPR / StCas9 Target Sites of the SCN9A Gene

[0533] The region of the SCN9A gene was scanned for target loci. Each region was scanned for a protospacer adjacent motif (PAM) with the sequence NNAGAAW. The gRNA 20bp spacer sequence corresponding to the PAM was then identified as shown in SEQ ID NO: 6251-8561 of the Sequence Listing.

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
Login to View More

PUM

No PUM Login to View More

Abstract

The present application provides materials and methods for treating a patient with one or more conditions associated with SCN9A whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and / or modulating the expression of SCN9A gene in a cell by genome editing.

Description

technical field [0001] The present disclosure relates to the field of gene editing, and in particular to the alteration of the voltage-gated sodium channel alpha subunit 9 (SCN9A) gene. [0002] related application [0003] This application claims the benefit of U.S. Provisional Application No. 62 / 358,763, filed July 6, 2016, and U.S. Provisional Application No. 62 / 461,874, filed February 22, 2017, both of which are incorporated by reference in their entirety into this article. [0004] Incorporated by reference into the sequence listing [0005] This application includes a sequence listing in computer readable form [Filename: 170152PCT(SCN9A) sequence listing (Part 1): 18,643,508 bytes – ASCII text file; created June 28, 2017; 170152PCT(SCN9A) sequence listing (Part 1) 2): 16,350,005 bytes – ASCII text file; created June 28, 2017; and 170152 PCT (SCN9A) sequence listing (Part 3): 14,922,048 bytes – ASCII text file; created June 28, 2017], which It is hereby incorporated b...

Claims

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
Login to View More

Application Information

Patent Timeline
no application Login to View More
Patent Type & Authority Applications(China)
IPC IPC(8): C07K14/705C12N9/22C12N15/10C12N15/11
CPCC12N15/86C12N15/113C07K14/47A61K48/0008A61K48/005A61K38/465A61K35/30A61P25/00A61P29/00A61P25/02C12N2750/14143C12N2310/20C07K14/705C12N15/1138C12N9/22A61P25/04A61P43/00C12N15/11C12N15/907C12N2800/80
Inventor A.S.伦德伯格S.库尔卡尼L.克莱因H.K.帕德马纳布汉
Owner VERTEX PHARMA INC
Who we serve
  • R&D Engineer
  • R&D Manager
  • IP Professional
Why Patsnap Eureka
  • Industry Leading Data Capabilities
  • Powerful AI technology
  • Patent DNA Extraction
Social media
Patsnap Eureka Blog
Learn More
PatSnap group products

Browse by: Latest US Patents, China's latest patents, Technical Efficacy Thesaurus, Application Domain, Technology Topic, Popular Technical Reports.

© 2024 PatSnap. All rights reserved.Legal|Privacy policy|Modern Slavery Act Transparency Statement|Sitemap|About US| Contact US: help@patsnap.com