Patent classifications
C12N2800/80
Single-vector type I vectors
The invention relates to the production and use of Cas-encoding sequences and vectors comprising these. Aspects of the invention provide products, vectors, delivery vehicles, uses and methods for producing Cas-encoding sequences in bacterial or archaeal cells.
Avoiding epigenetic silencing of exogenous nucleic acid in algae
The present application relates to the identification of novel DNA methyltransferases including CHG methylation in algal species. The present application relates to algal mutants permitting the expression of exogenous genes by alleviating the epigenetic mechanisms of CHG and CHH methylation of exogenous DNA and mono- and tri-methylation of lysine 9 of histone 3 (H3K9). This is achieved by mutating or attenuating the methyltransferase (MTase) genes in algae. The present application also relates to methods for efficiently expressing exogenous genes in algal species.
COMPOSITIONS AND METHODS FOR TREATING CEP290-ASSOCIATED DISEASE
Compositions and methods for treatment of CEP290 related diseases are disclosed.
FUSOSOME COMPOSITIONS FOR T CELL DELIVERY
The present disclosure provides, at least in part, methods and compositions for in vivo fusosome delivery. In some embodiments, the fusosome comprises a combination of elements that promote specificity for target cells, e.g., one or more of a fusogen, a positive target cell-specific regulatory element, and a non-target cell-specific regulatory element. In some embodiments, the fusosome comprises one or more modifications that decrease an immune response against the fusosome.
Compositions and Methods Comprising a TTR Guide RNA and a Polynucleotide Encoding an RNA-Guided DNA Binding Agent
Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.
METHODS FOR TARGETED CELL DEPLETION
Described herein are compositions, kits and methods for shredding the genomes of selected cell types, for example, the genomes of selected cancer cell types.
COMPOSITIONS COMPRISING A NUCLEASE AND USES THEREOF
The present invention relates to genes coding for nucleases, processes for characterizing the nucleases, cells comprising the nucleases, and methods of using the nucleases.
RETROTRANSPOSONS AND USE THEREOF
Systems and methods for targeted gene modification, targeted insertion, perturbation of gene transcripts, and nucleic acid editing. Novel nucleic acid targeting systems comprise components of CRISPR systems and non-LTR retrotransposon elements.
EFFICIENT TCR GENE EDITING IN T LYMPHOCYTES
The present disclosure relates to engineered T cells and methods of making and using the same, as well as reagents for making the engineered T cells.
Compositions, Methods and Systems for the Delivery of Gene Editing Material to Cells
This disclosure provides compositions, methods, and systems comprising a papillomaviral delivery vehicle for the delivery of gene editing material to cells. The papillomaviral delivery vehicle comprises a papillomavirus-derived capsid and DNA encoding a gene editing material encapsulated by the capsid. The papillomaviral delivery vehicle can be transduced into a cell under conditions conducive for the cell to synthesize the gene editing material. The cell can comprise a polynucleotide target and the gene editing material can target the polynucleotide target. The polynucleotide target can be a DNA polynucleotide target or RNA polynucleotide target.