C12N2750/14162

Restrictive inverted terminal repeats for viral vectors

This invention relates to modified parvovirus inverted terminal repeats (ITRs) that do not functionally interact with wild-type large Rep proteins, synthetic Rep proteins that functionally interact with the modified ITRs, and methods of using the same for delivery of nucleic acids to a cell or a subject. The modifications provide a novel Rep-ITR interaction that limits vector mobilization, increasing the safety of viral vectors.

RESTRICTIVE INVERTED TERMINAL REPEATS FOR VIRAL VECTORS
20230167418 · 2023-06-01 ·

This invention relates to modified parvovirus inverted terminal repeats (ITRs) that do not functionally interact with wild-type large Rep proteins, synthetic Rep proteins that functionally interact with the modified ITRs, and methods of using the same for delivery of nucleic acids to a cell or a subject. The modifications provide a novel Rep-ITR interaction that limits vector mobilization, increasing the safety of viral vectors.

A METHOD OF DETECTING AND/OR IDENTIFYING ADENO-ASSOCIATED VIRUS (AAV) SEQUENCES AND ISOLATING NOVEL SEQUENCES IDENTIFIED THEREBY
20170240921 · 2017-08-24 ·

Adeno-associated virus rh.20 sequences, vectors containing same, and methods of use are provided.

AAV VIRIONS WITH DECREASED IMMUNOREACTIVITY AND USES THEREFOR

Methods of making and using recombinant AAV virions with decreased immunoreactivity are described. The recombinant AAV virions include mutated capsid proteins or are derived from non-primate mammalian AAV serotypes and isolates that display decreased immunoreactivity relative to AAV-2.

ADENO-ASSOCIATED VIRAL VECTOR PRODUCER CELL LINES
20220177854 · 2022-06-09 ·

The invention relates to an adeno-associated virus (AAV) vector producer cell comprising nucleic acid sequences encoding AAV rep and cap genes, helper virus genes, and a DNA genome of the AAV vector; the AAV rep gene comprising an intron, the intron comprising a transcription termination 5 sequence with a first recombination site located upstream and a second recombination site located downstream of the transcription termination sequence; and the nucleic acid sequences all integrated together at a single locus within the AAV vector producer cell genome. The invention also relates to methods for producing the AAV vector producer cell lines.

AAV VIRIONS WITH DECREASED IMMUNOREACTIVITY AND USES THEREFOR

Methods of making and using recombinant AAV virions with decreased immunoreactivity are described. The recombinant AAV virions include mutated capsid proteins or are derived from non-primate mammalian AAV serotypes and isolates that display decreased immunoreactivity relative to AAV-2.

METHOD OF DETECTING AND/OR IDENTIFYING ADENO-ASSOCIATED VIRUS (AAV) SEQUENCES AND ISOLATING NOVEL SEQUENCES IDENTIFIED THEREBY
20220025401 · 2022-01-27 ·

Adeno-associated virus rh.20 sequences, vectors containing same, and methods of use are provided.

CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF

The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.

MODIFIED ADENO-ASSOCIATED VIRUS (AAV) PARTICLES FOR GENE THERAPY

The present invention relates to improved adeno-associated virus (AAV) particles for gene delivery and gene therapy. Provided are adeno associated virus (AAV) particles that comprise a modified capsid. The present invention further relates to methods for producing the improved AAV particles of this invention by removing natural binding sites in adeno associated virus (AAV) capsids and introducing ligand binding sites into said capsid to provide AAVs that transduce only particular cells of interest. An additional aspect of the present invention relates to modified AAV particles for use in the treatment of a disease and methods for treating a disease, comprising administering the modified AAV particles to a subject in need thereof. Yet a further aspect of this invention relates to the AAV particles of this invention for the transfection of cells, for example as a gene delivery tool in basic research.