Abstract: A recombinant adeno-associated virus (rAAV) vector comprising an AAVhu68 capsid produced in a production system comprising a nucleotide sequence of SEQ ID NO: 1, or a sequence at least 75% identical thereto which encodes SEQ ID NO:2. The AAVhu68 capsid comprises subpopulations of highly deamidated asparagine residues in asparagine-glycine pairs in the amino acid sequence of SEQ ID NO: 2. Also provided are compositions containing the rAAV and uses thereof. Additionally, rAAV having an engineered AAV capsid comprising at least one subpopulation of vp1 or vp2 proteins having a Val at amino acid position 157 with reference to the AAVhu68 vp1 numbering are provided.
Type:
Grant
Filed:
February 27, 2018
Date of Patent:
November 28, 2023
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
James M. Wilson, Qiang Wang, April Tepe, Kevin Turner
Abstract: Compositions and methods are provided for treating ocular disorders in a subject are provided. In one aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGA3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGB3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding REP-1. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.
Type:
Grant
Filed:
June 14, 2018
Date of Patent:
November 28, 2023
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
Jean Bennett, Junwei Sun, Jeannette Bennicelli
Abstract: Compositions and methods for treating metabolic diseases in a subject are provided. A viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding a GLP-1 receptor agonist fusion protein and regulatory sequences which direct expression thereof.
Type:
Application
Filed:
August 24, 2021
Publication date:
November 23, 2023
Applicant:
The Trustees of the University of Pennsylvania
Inventors:
James M. Wilson, Christian Hinderer, Makoto Horiuchi
Abstract: A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDS gene and two or more immunosuppressants is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating Hunter syndrome and the symptoms associated with Hunter syndrome.
Type:
Grant
Filed:
September 21, 2018
Date of Patent:
November 21, 2023
Assignee:
The Trustees of the University of Pennsylvania
Abstract: The presently disclosed subject matter provides a biomimetic lung disease model, and methods of its production and use. In one exemplary embodiment, the biomimetic lung disease model can include a first and second microchannel with a membrane coated with airway epithelial cells disposed between the microchannels and at least one device coupled to the biomimetic model that delivers an agent to at least one microchannel. In certain embodiments, the agent is cigarette smoke.
Type:
Grant
Filed:
July 27, 2016
Date of Patent:
November 14, 2023
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
Dongeun Huh, Mark Mondrinos, Woo Yul Byun
Abstract: De novo artificial protein based reporters that may be expressed in eukaryotic (e.g., mammalian) cells and methods of using the same are provided herein.
Type:
Grant
Filed:
June 29, 2021
Date of Patent:
November 14, 2023
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
Goutham Kodali, Molly Marie Sheehan, Joshua Mancini, Bohdana Marie Discher, Michael Magaraci, Nathan M. Ennist, Brian Chow, Peter Leslie Dutton, Christopher Moser
Abstract: The invention relates to compositions and methods for diagnosing as well as treating cancer diseases associated with choline kinase (ChoK). Specifically, the invention relates to a composition comprising an intrinsically fluorescent choline kinase (ChoK) inhibitor or a ChoK inhibitor operably linked to a fluorescent dye. The composition is capable of diagnosing and/or treating cancer diseases associated with ChoK.
Type:
Grant
Filed:
March 12, 2014
Date of Patent:
November 14, 2023
Assignee:
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
Inventors:
Edward J. Delikatny, Anatoliy V. Popov, Sean P. Arlauckas
Abstract: A recombinant vector comprises simian adenovirus 43, 45, 46, 47, 48, 49 or 50 sequences and a heterologous gene under the control of regulatory sequences. A cell line which expresses simian adenovirus 43, 45, 46, 47, 48, 49 or 50 gene(s) is also disclosed. Methods of using the vectors and cell lines are provided.
Type:
Grant
Filed:
October 29, 2019
Date of Patent:
November 7, 2023
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
Soumitra Roy, James M. Wilson, Luc H. Vandenberghe
Abstract: The present disclosure provides a device for imaging cardiac structures having at least a sheath, a wire-basket structure and an optical scope. The present disclosure also provides methods for imaging of epicardial space, epicardial ablation and cardiac ultrasound using the device disclosed herein.
Type:
Application
Filed:
June 29, 2023
Publication date:
November 2, 2023
Applicant:
The Trustees of the University of Pennsylvania
Abstract: The present invention relates to compositions and methods for treating diseases, disorders or conditions associated with the expression of the glycosyl-phosphatidylinositol (GPI)-linked GDNF family protein ?-receptor 4 (GFR?4).
Type:
Grant
Filed:
September 29, 2020
Date of Patent:
October 31, 2023
Assignees:
The Trustees of the University of Pennsylvania, The Scripps Research Institute
Inventors:
Donald L. Siegel, Vijay Bhoj, Christoph Rader, Rebecca Goydel
Abstract: Disclosed herein is a vaccine comprising a Middle East Respiratory Syndrome coronavirus (MERS-CoV) antigen. The antigen can be a consensus antigen. The consensus antigen can be a consensus spike antigen. Also disclosed herein is a method of treating a subject in need thereof, by administering the vaccine to the subject.
Type:
Grant
Filed:
September 10, 2021
Date of Patent:
October 31, 2023
Assignees:
The Trustees of the University of Pennsylvania, Inovio Pharmaceuticals, Inc.
Inventors:
David Weiner, Karuppiah Muthumani, Niranjan Y. Sardesai
Abstract: This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of RNA, oligoribonucleotide, and polyribonucleotide molecules.
Type:
Grant
Filed:
June 15, 2022
Date of Patent:
October 31, 2023
Assignee:
The Trustees of the University of Pennsylvania
Abstract: The present invention relates to Charcot-Marie-Tooth disease 2A (CMT2A) harboring the p.Arg364Trp or p.His361Tyr Mfn2 mutation, whose human counterpart results in severe, early-onset axonal neuropathy for p.Arg364Trp Mfn2 mutation in fertilized rat eggs. Cohorts of mutants and wild type littermates were characterized with multiple motor deficits that worsened over time. Separate cohorts of mutant and wild type at 7, 40, and 48 weeks showed reduced density of myelinated axons and active axonal degeneration in distal but not proximal nerves, as well as axonal degeneration in the fasciculus gracilis of the cervical spinal cord at 40 and 48 weeks not present in 7-week-old cohort Mfn2 mutants, or wild type at 7 or 40 weeks. The p.His361Tyr Mfn2 mutation using CRISPR/Cas9 showed abnormalities in gait dynamics at 8 weeks and lengthening of gait cycle at 16 weeks. The invention provides progressive axonal neuropathy for examining pathogenesis and treatment of CMT2A.
Type:
Grant
Filed:
October 30, 2017
Date of Patent:
October 31, 2023
Assignees:
Charcot-Marie-Tooth Association, Trustees of the University of Pennsylvania
Inventors:
Mark Albert Scheideler, Guojun Zhao, John Svaren, David C. Chan, Steven S. Scherer, Taleen Hanania
Abstract: The present application discloses compositions and methods for use of nucleoside modified mRNA that encode for at least one liver regenerative factor. The present invention also relates to compositions and methods for use of nucleoside modified mRNA complexed to nanoparticles. The disclosed compositions and methods are useful for treating acute liver diseases, chronic liver diseases, and/or acetaminophen (acetyl-para-aminophenol, APAP) overdose.
Type:
Application
Filed:
November 8, 2022
Publication date:
October 26, 2023
Applicants:
BOSTON MEDICAL CENTER CORPORATION, THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
Abstract: Provided herein are methods, systems and apparatus for diagnosing, predicting and/or preventing acute allograft vasculopathy in an organ transplant patient. The method comprises executing on a processor the steps comprising: analyzing clinical risk factors of a cardiac, kidney or liver transplant patient in a model which distinguishes between clinical biomarkers of allograft vasculopathy patients and non-allograft vasculopathy patients, and analyzing digital biopsy images from the transplant patient to morphologically differentiate patients which are prE allograft vasculopathy or which have active allograft vasculopathy from patients without acute allograft vasculopathy; assigning prE-, active or non-allograft vasculopathy status to a patient based on the outcome of the analysis of the clinical biomarkers and morphologic biomarkers; and where preE or active statis is assigned, treating the patient.
Type:
Application
Filed:
March 20, 2023
Publication date:
October 26, 2023
Applicants:
The Trustees of the University of Pennsylvania, Case Western Reserve University
Abstract: A composition, including a substrate having a planar array of depressions each defined by concave walls and a moat disposed around each depression of said array of depressions.
Type:
Grant
Filed:
June 26, 2015
Date of Patent:
October 24, 2023
Assignee:
The Trustees of the University of Pennsylvania
Abstract: A method for creating an irradiation plan. The method may include receiving an irradiation therapy plan for irradiating a target with a proton beam from a gantry. The irradiation therapy plan may have a plurality of irradiation spots for delivering protons to the target. The method may include identifying a rotational profile for angular rotation of the gantry with respect to the target, a slew time for a proton beam to travel between each of the plurality of irradiation spots, and an amount of protons to be delivered to each irradiation spot. The method may also include creating an irradiation plan. The irradiation plan may include a plurality of groups of irradiation spots based on the rotational profile, the slew time, and the amount of protons to be delivered to each irradiation spot. Each group of irradiation spots may be associated with a respective portion of the angular rotation.
Type:
Grant
Filed:
February 19, 2019
Date of Patent:
October 24, 2023
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
Alejandro Carabe-Fernandez, Peter Kimstrand
Abstract: Methods and compositions are provided for treatment of peroxisomal biogenesis disorders (PBDs). More particularly, recombinant adeno-associated viruses (rAAV) provided in the form of compositions are used to deliver a nucleic acid encoding human PEX1 to host cells. The rAAVs comprise a AAV capsid, and packaged therein a vector genome comprising an AAV 5? inverted terminal repeat (ITR) sequence; a promoter; a coding sequence encoding a human PEX1; and an AAV 3? ITR.
Type:
Grant
Filed:
May 31, 2018
Date of Patent:
October 24, 2023
Assignees:
The Trustees of the University of Pennsylvania, University of Southern California, The Research Institute of McGill University Health Centre
Inventors:
Nancy Braverman, Catherine Argyriou, Joseph Hacia, Jean Bennett, Junwei Sun, Ji Yun Song, Devin McDougald
Abstract: A microfluidic device contains a first layer having a plurality of channels, a second layer having a plurality of droplet makers, and a third layer having a plurality of through-holes connecting the plurality of channels to the plurality of droplet makers. The channels have a height of at least 4 times greater than the height of the droplet makers. The microfluidic device has at least 500 droplet makers in an area less than 10 cm2. The channels are formed by direct laser-micromachining and the droplet makers are formed by soft lithography molding.
Type:
Grant
Filed:
March 20, 2019
Date of Patent:
October 24, 2023
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
David Issadore, Melaku Muluneh Woldemariam