Abstract: The present invention relates to IL-8 inhibitor compounds, preferably dual CXCR1/CXCR2 receptor inhibitors, useful in the treatment and/or prevention of some sarcomas, preferably in the treatment and/or prevention of osteosarcoma, Ewing sarcoma, rhabdomyosarcoma or lung metastasis associated thereof.
Type:
Grant
Filed:
October 23, 2018
Date of Patent:
May 30, 2023
Assignees:
Dompe' Farmaceutici S.P.A., Research Institute at Nationwide Children's Hospital
Abstract: Provided herein are methods for preventing or treating antibiotic induced dysbiosis in a patient by administering to the subject a composition comprising a micro sphere, a bio film-generating probiotic bacterium and a prebiotic, wherein the prebiotic comprises a nutritional supplementation for the probiotic bacterium.
Type:
Application
Filed:
October 21, 2019
Publication date:
May 11, 2023
Applicant:
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Inventors:
Gail Besner, Michael Bailey, Steven David Goodman
Abstract: This disclosure provides isolated or recombinant polypeptides that are useful to vaccinate individuals suffering from chronic/recurrent biofilm disease or as a therapeutic for those with an existing infection. The individual's immune system will then naturally generate antibodies which prevent or clear these bacteria from the host by interfering with the construction and or maintenance of a functional protective biofilm. Alternatively, antibodies to the polypeptides can be administered to treat or prevent infection. Bacteria that cannot form functional biofilms are more readily cleared by the remainder of the host's immune system and/or traditional antibiotics.
Type:
Grant
Filed:
December 16, 2021
Date of Patent:
April 18, 2023
Assignee:
Research Institute of Nationwide Children's Hospital
Abstract: The present application provides materials and methods for treating a patient with a titin-based myopathy, particularly a titin-based cardiomyopathy, and/or other titinopathy. In addition, the present application provides materials and methods for editing the titin gene in a cell by genome editing.
Type:
Grant
Filed:
November 15, 2016
Date of Patent:
April 4, 2023
Assignee:
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Abstract: The present invention relates to methods and materials useful for systemically delivering polynucleotides across the blood brain barrier using adeno-associated virus as a vector. For example, the present invention relates to methods and materials useful for systemically delivering ?-N-acetylglucosamidinase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of these methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIB. As another example, the present invention relates to methods and materials useful for systemically delivering N-sulphoglucosamine sulfphohydrolase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of this second type of methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIA.
Abstract: Methods and materials for intrathecal delivery of recombinant Adeno-associated virus 9 (rAAV9) encoding Methyl-CpG binding protein 2 (MECP2) are provided. Use of the methods and materials is contemplated, for example, for the treatment of Rett syndrome.
Type:
Grant
Filed:
November 17, 2017
Date of Patent:
February 21, 2023
Assignees:
NATIONWIDE CHILDREN'S HOSPITAL, INC., OHIO STATE INNOVATION FOUNDATION
Abstract: The disclosure provides methods and compositions that are useful to lessen and/or cure bacterial biofilms and treat diseases or disorders associated with biofilms using one or more novel polypeptide vaccines, antibodies, antibody fragments and compositions. Bacteria that cannot form functional biofilms are more readily cleared by the remainder of the host's immune system and/or traditional antibiotics.
Type:
Grant
Filed:
January 3, 2018
Date of Patent:
January 31, 2023
Assignee:
Research Institute at Nationwide Children's Hospital
Abstract: It has been established that optimizing cell seeding onto tissue engineering vascular grafts (TEVG) is associated with reduced inflammatory responses and reduced post-operative stenosis of TEVG. Cell seeding increased TEVG patency in a dose dependent manner, and TEVG patency improved when more cells were seeded, however duration of incubation time showed minimal effect on TEVG patency. Methods of engineering patient specific TEVG including optimal numbers of cells to maintain graft patency and reduce post-operative stenosis are provided. Closed, single-use customizable systems for seeding TEVG are also provided. Preferably the systems are custom-designed based on morphology of the patient specific graft, to enhance the efficacy of cell seeding.
Type:
Grant
Filed:
December 12, 2016
Date of Patent:
January 3, 2023
Assignee:
Research Institute at Nationwide Children's Hospital
Inventors:
Christopher Breuer, Cameron Best, Robert Strouse, Narutoshi Hibino, Yong Ung-Lee
Abstract: 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.
Type:
Grant
Filed:
March 16, 2018
Date of Patent:
December 27, 2022
Assignee:
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Abstract: Provided herein are compositions comprising a biocompatible microsphere, a biofilm-generating probiotic bacterium, a prebiotic, and/or a prebiofilmic. Methods for preparing and formulating the compositions and methods for treating or preventing a disease using the compositions are also provided.
Type:
Grant
Filed:
March 16, 2020
Date of Patent:
November 15, 2022
Assignees:
Research Institute at Nationwide Children's Hospital, Ohio State Innovation Foundation
Inventors:
Steven D. Goodman, Lauren O. Bakaletz, Michael Bailey, Gail Besner
Abstract: The present disclosure relates to a novel CRISPR-Cas9 based system for editing mitochondrial DNA. Aspects of the disclosure provide for mitochondrial translocation of both the guide RNA and the recombinant Cas9 nuclease.
Type:
Grant
Filed:
November 15, 2017
Date of Patent:
November 8, 2022
Assignee:
Research Institute at Nationwide Children's Hospital
Inventors:
Kenneth John McLaughlin, Syed-Rehan Ashfaq Hussain
Abstract: Provided herein are microspheres containing an antimicrobial agent encapsulated within a glycosidic polymer and use thereof, especially to inhibit the growth of carie-causing organism.
Type:
Grant
Filed:
June 11, 2019
Date of Patent:
November 1, 2022
Assignee:
Research Institute at Nationwide Children's Hospital
Inventors:
Steven David Goodman, Lauren Mashburn Warren
Abstract: The present invention relates to recombinant adeno-associated virus (rAAV) delivery of an alpha-sarcoglycan gene. The invention provides rAAV products and methods of using the rAAV in the treatment of limb girdle muscular dystrophies such as LGMD2D.
Type:
Grant
Filed:
September 19, 2018
Date of Patent:
September 20, 2022
Assignee:
NATIONWIDE CHILDREN'S HOSPITAL, INC.
Inventors:
Jerry R. Mendell, Louis Chicoine, Louise Rodino-Klapac, Kelly Reed Clark, Thomas J. Preston
Abstract: Recombinant polynucleotides and vectors containing an engineered (artificial) exon-intron-exon gene structure in a transgene are provided, which undergoes splicing when it is expressed in a target cell.
Type:
Application
Filed:
February 19, 2020
Publication date:
August 18, 2022
Applicant:
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Inventors:
Timothy P. Cripe, Chun-yu Chen, Brian Hutzen, Mark Currier, Pin-yi Wang, Dawn Chandler
Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-osmolar contrast agent, together with a rAAV9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.
Type:
Grant
Filed:
May 8, 2019
Date of Patent:
August 16, 2022
Assignees:
NATIONWIDE CHILDREN'S HOSPITAL, OHIO STATE INNOVATION FOUNDATION
Inventors:
Brian K. Kaspar, Arthur Burghes, Paul Porensky
Abstract: 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 dystrophinopathy or muscular dystrophy. The invention also provides for combination therapies comprising expressing both miR-29 and micro-dystrophin to reduce and prevent fibrosis in patients suffering from dystrophinopathy or muscular dystrophy.
Type:
Grant
Filed:
April 14, 2017
Date of Patent:
August 9, 2022
Assignee:
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Inventors:
Louise Rodino-Klapac, Jerry R. Mendell, Kristin N. Heller
Abstract: Described herein are recombinant AAV vectors comprising a polynucleotide sequence comprising ?-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 ?-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.
Type:
Grant
Filed:
April 14, 2017
Date of Patent:
June 14, 2022
Assignee:
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Abstract: The present invention relates to a tissue-specific promoter system for expressing microRNA (miRNA) for RNA interference-based methods of gene therapy. In these systems, the miRNA will inhibit gene expression or replace natural miRNA expression using microRNA.
Type:
Grant
Filed:
March 31, 2017
Date of Patent:
May 31, 2022
Assignee:
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Abstract: The present invention provides novel methods and kits for determining whether a subject is suffering from endometriosis or an associated disorder. It also provides methods and kits for determining whether a subject suffering from endometriosis or an associated disorder is more or less likely to be responsive to a therapy to treat endometriosis or an associated disorder.
Type:
Application
Filed:
July 2, 2021
Publication date:
May 26, 2022
Applicant:
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Inventors:
Li Chen, Yu Zhou, David Brigstock, Gail Besner
Abstract: The disclosure provides a non-natural herpes simplex virus (“HSV”), compositions comprising, or alternatively consisting essentially of, or yet further consisting of the HSV, and methods of producing the HSV, or infecting a cell with the HSV. Also provided herein are methods of treating cancer or inhibiting the growth or metastasis of cancer cell in a subject in need thereof.
Type:
Application
Filed:
March 13, 2020
Publication date:
May 19, 2022
Applicant:
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Inventors:
Timothy P. Cripe, Kevin A. Cassady, Pin-Yi Wang, Julia K. Halley