City of Hope

University of California

Mayo Foundation for Medical Education and Research

John C Burnett

John J Rossi

Elizabeth Epps

Horng H Chen

Robert D Simari

Shuchong Pan

Anh Pham

Candace Y Lee

Compositions and methods for mitochondria genome editing are provided. Also provided are methods for treating mitochondrial disorders by the disclosed compositions.

Compositions and methods for mitochondrial genome editing

Provided herein are, inter alia, oligonucleotides, kits, and methods useful for increasing lentiviral titers.

Splice inhibiting oligonucleotides

MicroRNAs embedded within an intron, which are called 'mirtrons,' can be used as a platform for expressing one or more shRNA or miRNA mimics in a lentiviral vector. The inventors developed a strategy to improve lentiviral titering by reducing the production of shRNA/miRNA from the vector during packaging through the introduction of splice-inhibiting antisense oligonucleotides during vector packaging, which inhibit the splicing of the mirtron and subsequent processing of the shRNAs/miRNAs. In an aspect is provided a kit comprising an oligonucleotide comprising a mirtron splice site binding sequence and a lentiviral packaging system. In an aspect is provided a method for producing a lentivirus. The method comprises the step of transfecting a cell with an oligonucleotide comprising a mirtron splice site binding sequence and a lentiviral packaging system; thereby producing the lentivirus.

Growing community of inventors

Chatsworth, CA, United States of America

John C Burnett