Optimized Virus-like Particles for Cas9 RNPs & Transgene/HDR Template Delivery
Tech ID: 32400 / UC Case 2021-177-0
Patent Status
Patent Pending
Brief Description
The inventors have developed optimized methods for using virus-like particles for the co-delivery of Cas9 ribonucleoprotein complexes and:
- a lentiviral genome that encodes a large transgene, such as a chimeric angtigen receptor (CAR) transgene
- a lentiviral genome that does not encode a sgRNA expression cassette
- a method for nucleofecting VLPs + homology directed repair (HDR) donor template together to enhance HDR in treated cells
Suggested uses
In vivo/ex vivo generation of CRISPR-Cas9 gene edited CAR T cells.
Any application where a simultaneous gene knockout and transgene introduction/expression is desired.
Advantages
This technology offers a one-step strategy using engineered lentiviral particles to introduce Cas9 RNPs and a CAR transgene into primary human T cells without electroporation. Furthermore, programming particle tropism allowed the inventors to target a specific cell type within a mixed cell population. This adaptable approach to immune cell engineering ex vivo provides a strategy applicable to genetic modification of targeted somatic cells in vivo.
Contact
- Terri Sale
- terri.sale@berkeley.edu
- tel: View Phone Number.
Inventors
- Doudna, Jennifer A.
Other Information
Additional Technologies by these Inventors
- Methods and Compositions for Using Argonaute to Modify a Single-Stranded Target Nucleic Acid
- COMPOSITIONS AND METHODS FOR IDENTIFYING HOST CELL TARGET PROTEINS FOR TREATING RNA VIRUS INFECTIONS
- Cas9 Variants With Altered DNA Cleaving Activity
- 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
- Methods For High Signal-To-Noise Imaging Of Chromosomal Loci In Cells Using Fluorescent Cas9
- A Dual-RNA Guided CasZ Gene Editing Technology
- MODULATORS OF TYPE VI-D CRISPR-CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF
- CRISPR-CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF (“Cas-VariPhi”)
- A Protein Inhibitor Of Cas9
- Small Cas9 Protein Inhibitor
- Split-Cas9 For Regulatable Genome Engineering
- Decorating Chromatin for Precise Genome Editing Using CRISPR
- CRISPR-CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF (“Cas-Theta”)
- COMPOSITIONS AND METHODS FOR INCREASING HOMOLOGY-DIRECTED REPAIR
- CRISPR CASY COMPOSITIONS AND METHODS OF USE
- Single Conjugative Vector for Genome Editing by RNA-guided Transposition
- CRISPR-CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF (“Cas-Omega”)
- CRISPR-CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF
- Engineered/Variant Hyperactive CRISPR CasPhi Enzymes And Methods Of Use Thereof
- 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 (CasGamma)
- 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
- Chimeric Cas9 Variants With Novel Engineered Enzymatic Activities
- Small Molecule Assisted Cell Penetrating Cas9 RNP Delivery
- Methods and Compositions for Controlling Gene Expression by RNA Processing
