University of Iowa Research Foundation

Massachusetts Institute of Technology

The Broad Institute, Inc.

Editas Medicine, Inc.

University of Florida Research Foundation, Incorporated

Edgardo Rodriguez

Beverly L Davidson

Feng Zhang

Jordi Mata-Fink

Alexis Borisy

Deborah Palestrant

Chie-yu Lin

Sergei Zolotukhin

William W Hauswirth

Nicholas Muzyczka

Barry John Byrne

Alejandro Mas Monteys

Richard O Snyder

Terence R Flotte

Chie-Yu Lin

Mark R Potter

Yoshihisa Sakai

Irine Zolotukhin

Scott Loiler

Vince A Chiodo

Corinna Burger

Kevin R Nash

Thomas J Fraites

The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences especially for use as to nucleotide repeat disorders. Provided are delivery systems and tissues or organ which are targeted as sites for delivery especially for use as to nucleotide repeat disorders. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex or system especially for use as to nucleotide repeat disorders, as well as methods for the design and of such. Also provided are methods of directing CRISPR complex or system formation in eukaryotic cells especially for use as to nucleotide repeat disorders including with consideration of specificity for target recognition and avoidance of toxicity and editing or modifying a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders

The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a SIN CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing SIN CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

Disclosed herein are methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas9 molecule/gRNA complex.

CRISPR-related methods and compositions with governing gRNAS

CRISPR-related methods and compositions with governing gRNAs

An isolated promoter sequence comprising a nucleic acid of between 600 and 1700 nucleotides in length having at least 90% identity to SEQ ID NO:1, SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4, SEQ ID NO:5, SEQ ID NO:6, or SEQ ID NO:7.

Promoter compositions

Vectors that encode Adeno-Associated Virus (AAV) Rep and Cap proteins of different serotypes and Adenovirus transcription products that provide helper functions were used to produce pseudotyped recombinant AAV (rAAV) virions. Purification methods generated pseudotyped rAAV virion stocks that were 99% pure with titers of 1&#xd7;10&#x2013;1&#xd7;10vector genomes/ml.

Production of pseudotyped recombinant AAV virions

Growing community of inventors

Gainesville, FL, United States of America

Edgardo Rodriguez