Abstract: Systems and methods for improved cranio-maxillo-facial distraction are provided. The disclosed subject matter can include a distraction device adapted for subcutaneous implantation in a patient, and a handheld device operationally coupled to the distraction device. The distraction device can include a distraction element, adapted to attach to opposing bony fragments of a bone and perform distraction osteogenesis on the bone, and a rotatable magnetic element, coupled to the distraction element such that rotation of the magnetic element controls the distraction element. The handheld device can include a magnetic element, operationally coupled to and magnetically aligned with the magnetic element of the distraction device, such that rotation of the magnetic element of the handheld device causes corresponding rotation of the magnetic element of the distraction device.
Type:
Application
Filed:
February 12, 2020
Publication date:
April 28, 2022
Applicants:
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA, CHILDREN'S HOSPITAL OF PHILADELPHIA
Abstract: Provided are compositions and methods for inducing expression of human beta-globin in erythrocytes for use in prophylaxis and/or therapy of a hemoglobinopathy in an individual. The method generally entails introducing into CD34+ cells a poly-nucleotide encoding: i) a 5? long terminal repeat (LTR) and a self-inactivating 3? LTR; ii) at least one polyadenylation signal; iii) at least one promoter; iv) a globin gene locus control region (LCR); v) an ankyrin insulator element (Ank); vi) a Woodchuck Post-Regulatory Element (WPRE) configured such that the WPRE does not integrate into a target genome; and vii) a sequence that is a reverse complement of a sequence encoding human beta-globin, and can include beta-globin that has a PT87Q mutation. Intron 2 of the beta globin gene can be a complete intron. Modified erythrocyte progenitor cells, recombinant vectors and virions comprising recombinant polynucleotides, and methods of making the vectors and virions are included.
Type:
Grant
Filed:
January 21, 2016
Date of Patent:
April 26, 2022
Assignees:
CORNELL UNIVERSITY, THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Inventors:
Stefano Rivella, Laura Breda, Alisa Dong, Gerd Blobel, Wulan Deng
Abstract: Methods and uses for diagnosing and treating anxiety disorders are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.
Type:
Application
Filed:
October 28, 2021
Publication date:
April 21, 2022
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Abstract: Methods for diagnosing and treating conduct disorder are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.
Type:
Application
Filed:
October 27, 2021
Publication date:
April 21, 2022
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Abstract: Methods and uses for diagnosing and treating Tourette syndrome are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.
Abstract: Compositions and methods for AAV mediated gene therapy are disclosed. AAV vectors comprise protein capsid variants comprising therapeutically beneficial transgenes. AAV variants are provided which exhibit increased transduction efficiency when compared to AAV serotypes (e.g., AAV1, AAV2, AAV8, AAV-rh74), which lack the modifications disclosed herein. Such improved vectors are useful for transduction of a variety of tissues.
Type:
Grant
Filed:
February 2, 2018
Date of Patent:
March 22, 2022
Assignee:
The Children's Hospital of Philadelphia
Inventors:
Mustafa N. Yazicioglu, Federico Mingozzi, Xavier Anguela, Katherine A. High
Abstract: The present invention provides compositions and methods for treating cancer in a patient. In one embodiment, the method comprises a first-line therapy comprising administering to a patient in need thereof a genetically modified T cell expressing a CAR wherein the CAR comprises an antigen binding domain, a transmembrane domain, a costimulatory signaling region, and a CD3 zeta signaling domain and monitoring the levels of cytokines in the patient post T cell infusion to determine the type of second-line of therapy appropriate for treating the patient as a consequence of the presence of the CART cell in the patient.
Type:
Grant
Filed:
April 26, 2018
Date of Patent:
March 15, 2022
Assignees:
The Trustees of the University of Pennsylvania, The Children's Hospital of Philadelphia
Inventors:
Carl H. June, Bruce L. Levine, Michael D. Kalos, Stephan Grupp
Abstract: Provided are macromolecular prodrugs in which camptothecin analogs are covalently bonded to polymers via ester bonds that are labile under physiological conditions. Also provided are methods of treating cancer, especially neuroblastoma with the macromolecular prodrugs.
Type:
Grant
Filed:
September 17, 2019
Date of Patent:
February 22, 2022
Assignee:
The Children's Hospital of Philadelphia
Inventors:
Michael Chorny, Ivan Alferiev, Garrett M. Brodeur
Abstract: Methods for diagnosing and treating autism spectral disorders are encompassed. In one embodiment, a patient is diagnosed as having autism spectral disorder if at least one CNV in an mGluR network gene is found in a patient sample. Patients with at least one mGluR network gene CNV are effectively treated with (+)-5-oxo-Dprolinepiperidinamide monohydrate (NS-105).
Abstract: The present disclosure provides improved compositions and methods for T-cell-based immunotherapy employing a modified T cells obtained from the lymphatic system of normal and cancer patient donors. These cells can then be used to treat patients for a variety of different blood and solid tumor cancers.
Type:
Application
Filed:
October 30, 2019
Publication date:
December 30, 2021
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Abstract: This disclosure is directed to methods of methods of computing a composite measurement comprising employing two or more sub-instruments to measure one or more clinical symptoms of mitochondrial dysfunction or mitochondrial disease. This disclosure is also directed to methods of assessing and managing a subject with mitochondrial dysfunction or mitochondrial disease using a composite measurement.
Type:
Application
Filed:
June 26, 2019
Publication date:
December 2, 2021
Applicants:
RIBONOVA INC., The Children's Hospital of Philadelphia
Inventors:
Marni J. Falk, Nigel L. Webb, Shana E. McCormack, Eric C. Yuen, Zarazuela Zolkipli-Cunningham, Anthony W. Ford-Hutchinson
Abstract: An auricular support system can include a substrate and a mold material. The substrate can include a substrate body having a first end and an opposed second end. At least a portion of the first end can be configured to carry an adhesive suitable to attach to a dermal surface. The second end defines at least one void. The mold material can be configured to be applied to both an auricular structure and the second end of the substrate body such that the mold material becomes disposed in the void and molds about the auricular structure. The mold material can be configured to cure after the mold material is applied to the auricular structure and the substrate body so as to support the auricular structure relative to the substrate. The auricular support system can further include an auxiliary support structure configured to attach to the auricular structure.
Abstract: Methods for diagnosing and treating conduct disorder are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.
Abstract: Provided herein are chimeric transactivator minigenes, where the alternative splicing of the minigene determines whether a transactivator is expressed. Expression of the transactivator results in the transcription of a target gene that is under the control of a designer promoter sequence. Alternatively, provided herein are chimeric target gene minigenes, wherein the alternative splicing of the minigene directly determines whether the target gene is expressed. The target gene may encode an inhibitory RNA, a CRISPR-Cas9 protein, or a therapeutic protein.
Type:
Application
Filed:
August 7, 2019
Publication date:
November 18, 2021
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Inventors:
Beverly L. DAVIDSON, Alejandro Mas MONTEYS, Ammiel Al HUNDLEY, Paul T. RANUM
Abstract: Provided herein are engineered red blood cells expressing rare blood antigen group profiles, and methods of making use the same, are described. Also provided are recombinant reagent red blood cells that express or lack the expression of at least one protein (e.g., a blood group antigen) on its surface and uses thereof.
Type:
Application
Filed:
October 29, 2018
Publication date:
November 18, 2021
Applicants:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA, NEW YORK BLOOD CENTER, INC.
Abstract: Methods and uses for diagnosing and treating anxiety disorders are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.
Abstract: The invention relates to modified Factor IX coding sequence, expression cassette, vectors such as viral (e.g., lenti- or adeno-associated viral) vectors, and gene transfer methods and uses. In particular, to target Factor IX nucleic acid to cells, tissues or organs for expression (transcription) of Factor IX.
Type:
Application
Filed:
July 12, 2021
Publication date:
October 28, 2021
Applicant:
The Children's Hospital of Philadelphia