Small Molecule Assisted Cell Penetrating Cas9 RNP Delivery
Patent Status
| Country | Type | Number | Dated | Case |
| United States Of America | Published Application | 20200115688 | 04/16/2020 | 2017-098 |
Brief Description
Clustered regularly interspaced short palindromic repeats (CRISPR)
Cas systems provide a means for modifying genomic information and have the
potential to revolutionize the treatment of genetic diseases. Although RNA-programmed
Cas9 has proven to be a versatile tool for genome engineering in multiple cell types
and organisms, it has been challenging to develop the therapeutics because they
require the simultaneous in vivo delivery of the Cas9 protein, guide RNA and
donor DNA. Compositions that can increase the efficiency of such delivery,
particular in eukaryotic cells, are greatly needed.
UC Researchers have discovered that the inclusion of an
agent that decreases the acidity of an endosome inside eukaryotic cells, in a
genome editing composition, increased the efficiency of genome editing. The agent was included in a composition having
an RNA-guided endonuclease and an RNA-guided endonuclease and was used for gene
editing.
Suggested uses
- Genome editing (particularly in eukaryotic cells)
- Research reagent
- Gene therapy
- Increased Delivery of CRISPR Cas components to eukaryotic cells
Advantages
- Significantly increased gene editing efficiency through increased delivery through endosome
Contact
- Terri Sale
- terri.sale@berkeley.edu
- tel: View Phone Number.
Inventors
- Doudna, Jennifer A.
Other Information
Keywords
CRISPR, delivery, eukaryotic cells
Additional Technologies by these Inventors
- COMPOSITIONS AND METHODS FOR IDENTIFYING HOST CELL TARGET PROTEINS FOR TREATING RNA VIRUS INFECTIONS
- Lentivirus-like Particle Delivery of CRISPR-Cas9 & Guide RNA for Gene Editing
- Genome Editing via LNP-Based Delivery of Efficient and Stable CRISPR-Cas Editors
- Type III CRISPR-Cas System for Robust RNA Knockdown and Imaging in Eukaryotes
- Cas12-mediated DNA Detection Reporter Molecules
- Improved guide RNA and Protein Design for CasX-based Gene Editing Platform
- Cas13a/C2c2 - A Dual Function Programmable RNA Endoribonuclease
- RNA-directed Cleavage and Modification of DNA using CasY (CRISPR-CasY)
- CasX Nickase Designs, Tans Cleavage Designs & Structure
- A Dual-RNA Guided CasZ Gene Editing Technology
- CRISPR-CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF (“Cas-VariPhi”)
- Modifications To Cas9 For Passive-Delivery Into Cells
- A Protein Inhibitor Of Cas9
- RNA-directed Cleavage and Modification of DNA using CasX (CRISPR-CasX)
- Compositions and Methods for Genome Editing
- Split-Cas9 For Regulatable Genome Engineering
- NANOPORE MEMBRANE DEVICE AND METHODS OF USE THEREOF
- CRISPR CASY COMPOSITIONS AND METHODS OF USE
- Single Conjugative Vector for Genome Editing by RNA-guided Transposition
- Improved Cas12a Proteins for Accurate and Efficient Genome Editing
- CRISPR-CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF
- Engineered/Variant Hyperactive CRISPR CasPhi Enzymes And Methods Of Use Thereof
- Engineering Cas12a Genome Editors with Minimized Trans-Activity
- Methods Of Use Of Cas12L/CasLambda In Plants
- Type V CRISPR/CAS Effector Proteins for Cleaving ssDNA and Detecting Target DNA
- THERMOSTABLE RNA-GUIDED ENDONUCLEASES AND METHODS OF USE THEREOF (GeoCas9)
- Structure-Guided Methods Of Cas9-Mediated Genome Engineering
- Endoribonucleases For Rna Detection And Analysis
- Efficient Site-Specific Integration Of New Genetic Information Into Human Cells
- CRISPR-Cas Effector Polypeptides and Methods of Use Thereof
- Class 2 CRISPR/Cas COMPOSITIONS AND METHODS OF USE
- Compositions and Methods of Use for Variant Csy4 Endoribonucleases
- Identification Of Sites For Internal Insertions Into Cas9
- Methods and Compositions for Controlling Gene Expression by RNA Processing
