Jerry R Mendell

20110301226, Dec 8, 2011

Dec 2, 2009

13/132783

Joshua T. Mendell - Dallas TX, US
Kathryn A. Mendell - Baltimore MD, US
Raghu R. Chivukula - Baltimore MD, US
Erik A. Wentzel - Leesburg VA, US
Jerry R. Mendell - Columbus OH, US
K. Reed Clark - Westerville OH, US
Janaiah Kota - Columbus OH, US

Nationwide Children's Hospital, Inc - Columbus OH
THE JOHNS HOPKINS UNIVERSITY - Baltimore MD

A61K 48/00
C12N 15/85
C12N 15/86
A61P 1/16
C12N 5/10
C12Q 1/68
A61P 35/00
C12N 5/09
C12N 15/861

514 44 R, 435375, 4353201, 435325, 435 611, 435 613

The present invention provides compositions and methods featuring miR-26 microRNA polynucleotides for the diagnosis, treatment or prevention of hepatic neoplasia.

20100178348, Jul 15, 2010

Nov 29, 2007

12/516995

Brian K. Kaspar - Westerville OH, US
Jerry R. Mendell - Columbus OH, US

Nationwide Children's Hospital - Columbus OH

A61K 9/14
A61K 35/76
C07H 21/00
C12N 7/01
C12N 5/071
C07H 21/04
A61P 21/00

424489, 424 932, 536 2372, 4352351, 435325

The present invention relates to methods for inhibiting myostatin, a regulator of muscle mass, for muscle enhancement (including inducing hypertrophy and/or hyperplasia) as well as improving muscle function (including decreasing atrophy and/or increasing endurance, force and/or strength). The methods involve delivering genes to cells using gene delivery in order to inhibit myostatin. Examples of genes to be delivered are genes encoding’ proteins such as Follistatin, Follistatin-related gene-1 (FLRG-I), growth differentiation factor associated protein-1 (GASP-I) and myostatin precursor propeptide. The genes are delivered using a recombinant Adeno-associated virus (rAAV) lacking rep and cap DNA capable of infecting the cells. Following introduction, the genes are expressed in the cell body of the infected cell and the encoded proteins are secreted systemically. In other methods-of the invention expression of proteins such as activin lib and myostatin is inhibited by oligonucleotide techniques to effect muscle enhancement. All the methods have applications in the treatment of musculoskeletal and neurodegenerative disorders among others, as well as enhancing muscle in livestock.

20220364117, Nov 17, 2022

Jun 3, 2022

17/832325

- Columbus OH, US
Jerry R. Mendell - Columbus OH, US

C12N 15/86
A61P 21/00
A61K 9/00
A61K 38/17
C12N 7/00

The invention provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscles including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.

20220290180, Sep 15, 2022

Aug 21, 2020

17/635978

- Columbus OH, US
Danielle Griffin - Canal Winchester OH, US
Jerry R. Mendell - Columbus OH, US

C12N 15/86
C07K 14/47

Described herein are methods of treating muscular dystrophy in a subject, comprising administration of a recombinant AAV vector AAVrh74.tMCK.hSCGA using a systemic route of administration and at a dose of about 1.0×10vg/kg to about 5.0×10vg/kg. Further disclosed are methods of expressing alpha-sarcoglycan gene in a cell or in a subject in need thereof, decreasing a serum CK level, and increasing alpha-sarcoglycan positive fibers in muscle tissue of a subject.

20220251156, Aug 11, 2022

Jan 20, 2022

17/580228

- Columbus OH, US
Jerry R. Mendell - Columbus OH, US

C07K 14/47
A61K 48/00
C07K 14/705
C12N 15/86
A61P 25/14
A61P 21/00
C12N 15/85

Described herein are recombinant AAV vectors comprising a polynucleotide sequence comprising β-sarcoglycan and methods of using the recombinant vectors to reduce or prevent fibrosis in a mammalian subject suffering from a muscular dystrophy. Also described herein are combination therapies comprising administering AAV vector(s) expressing β-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.

20230001015, Jan 5, 2023

Apr 12, 2022

17/719019

- Columbus OH, US
Jerry R. Mendell - Columbus OH, US
Kristin N. Heller - Columbus OH, US

A61K 48/00
C07K 14/47
A61K 38/17
A61K 31/7088
C12N 15/11
C12N 15/113
C12N 15/86
A61P 21/00
A61K 9/00
A61P 19/04
A61K 35/761
C12N 15/864

The invention provides for recombinant AAV vectors comprising a polynucleotide sequence comprising the guide strand of miR-29c and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.

20210260218, Aug 26, 2021

Jun 17, 2019

17/253956

- Columbus OH, US
Jerry R. Mendell - Columbus OH, US

A61K 48/00
C12N 15/86
C07K 14/47
A61P 21/00

The invention provides gene therapy vectors, such as adeno-associated vims (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscle s including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.

20210128749, May 6, 2021

Mar 16, 2018

16/757207

- Columbus OH, US
Jerry R. Mendell - Columbus OH, US

A61K 48/00
C12N 15/86
A61P 21/00

The invention provides gene therapy vectors, such as adeno-associated vims (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscle including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.