Tissue-Specific Genome Engineering Using CRISPR-Cas9
Tech ID: 25143 / UC Case 2015-205-0
Patent Status
| Country | Type | Number | Dated | Case |
| Japan | Issued Patent | 7268103 | 04/24/2023 | 2015-205 |
| United States Of America | Issued Patent | 10,851,367 | 12/01/2020 | 2015-205 |
| Japan | Published Application | P2018-537448A | 12/20/2018 | 2015-205 |
| European Patent Office | Published Application | 3 373 979 | 09/19/2018 | 2015-205 |
| Argentina | Published Application | AR 106639 A1 | 02/07/2018 | 2015-205 |
Additional Patents Pending
Brief Description
Delivering gene-editing agents safely and effectively has long been a challenge in modern medicine. Traditional methods using viral vectors introduce risks such as insertional mutagenesis, hepatotoxicity, and transient therapeutic effects due to immune responses. UC Berkeley researchers have developed a groundbreaking tissue-specific genome engineering system utilizing CRISPR-Cas9, offering a safer and more precise alternative for gene therapy.
UC Berkeley researchers and others have created compounds, compositions, uses thereof the combines the cutting-edge CRISPR technology with advanced targeting mechanishms for the treatment of diseases, conditions and/or disorders, and uses thereof as asialoglycoprotein receptor (ASGPR) targeting agents.
Suggested uses
- Gene editing
- Gene delivery
Advantages
- By leveraging ASGPR targeting agents, this approach ensures precise uptake by hepatocytes and other specific tissues.
- Avoids the risks associated with viral vectors, reducing toxicity and immune complications.
- By delivering gene-editing agents in protein form rather than DNA or RNA, the technology maximizes therapeutic potential while minimizing unwanted genetic disruptions.
- Overcomes the limitations of receptor-mediated endocytosis to ensure the therapeutic agents effectively reach their intended subcellular locations.
Contact
- Terri Sale
- terri.sale@berkeley.edu
- tel: View Phone Number.
Inventors
- Doudna, Jennifer A.
Other Information
Categorized As
Additional Technologies by these Inventors
- COMPOSITIONS AND METHODS FOR IDENTIFYING HOST CELL TARGET PROTEINS FOR TREATING RNA VIRUS INFECTIONS
- 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
- Compositions and Methods for Delivering Molecular Cargo to Cells
- Cas13a/C2c2 - A Dual Function Programmable RNA Endoribonuclease
- Miniature Type VI CRISPR-Cas Systems and Methods of Use
- RNA-directed Cleavage and Modification of DNA using CasY (CRISPR-CasY)
- CasX Nickase Designs, Tans Cleavage Designs & Structure
- In Vivo Gene Editing Of Tau Locus Via Liponanoparticle Delivery
- Methods and Compositions for Modifying a single stranded Target Nucleic Acid
- A Dual-RNA Guided CasZ Gene Editing Technology
- CRISPR-CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF (“Cas-VariPhi”)
- A Protein Inhibitor Of Cas9
- RNA-directed Cleavage and Modification of DNA using CasX (CRISPR-CasX)
- Compositions and Methods for Genome Editing
- IS110 and IS1111 Family RNA-Guided Transposons
- Methods to Interfere with Prokaryotic and Phage Translation and Noncoding RNA
- Variant Cas12a Protein Compositions and Methods of Use
- In Vitro and In Vivo Genome Editing by LNP Delivery of CRISPR Ribonucleoprotein
- 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
- 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)
- Variant TnpB and wRNA Proteins
- Structure-Guided Methods Of Cas9-Mediated Genome Engineering
- Efficient Site-Specific Integration Of New Genetic Information Into Human Cells
- Class 2 CRISPR/Cas COMPOSITIONS AND METHODS OF USE
- Compositions and Methods of Use for Variant Csy4 Endoribonucleases
- Methods and Compositions for Controlling Gene Expression by RNA Processing
