Abstract: This disclosure relates to the identification of a subset of mGluR network gene CNVs that are predictive of efficacy of treatment with fasoracetam, as well as the identification of an mGluR network gene CNV that is predictive of an increased likelihood of having ADHD as well as having certain symptoms associated with ADHD.
Type:
Application
Filed:
June 7, 2023
Publication date:
May 16, 2024
Applicant:
The Children's Hospital of Philadelphia
Inventors:
Garry A. Neil, Liza Squires, Hakon Hakonarson
Abstract: An enema assist device is provided. The device includes a support and the support is configured to be placed on a toilet bowl such that the support front is oriented towards the front of the toilet bowl. The support is configured to support a fluid delivery tip such that the fluid delivery tip is disposed on the top surface of the support and protrudes at least partially beyond a plane defined by a top of the toilet seat.
Type:
Application
Filed:
March 22, 2022
Publication date:
May 16, 2024
Applicant:
The Children's Hospital of Philadelphia
Inventors:
Joy Kerr, Michelle Hsia, Jason Van Batavia
Abstract: A system having patient image processing capability and being in compliance with health insurance portability and accountability act including a N-server having a database, wherein the N-server receives patient data in a form of numerical values from at least one sender and wherein the patient data is converted into numerical values prior to being sent to the N-server. Additionally, the system includes one or more artificial intelligence program to analyze the data in the form of numerical values and detect patterns for a predefined abnormality.
Type:
Grant
Filed:
February 20, 2020
Date of Patent:
May 7, 2024
Assignee:
CHILDREN'S HOSPITAL LOS ANGELES
Inventors:
Fernando Yepes Calderon, James Gordon McComb
Abstract: A strong insulator fragment from foamy virus, which can be used to insulate expression of a transgene and reduce genotoxicity of integrating vectors comprising such. The insulator fragment can also be used in gene targeting constructs in gene editing.
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:
December 19, 2023
Publication date:
April 25, 2024
Applicant:
The Children's Hospital of Philadelphia
Abstract: Activity-inducible fusion proteins whose activity is post-translationally regulated utilizing a hsp90 binding domain and a drug molecule are described. In the absence of the drug molecule, the activity-inducible fusion proteins are inactivated but can be activated by a relevant physiological parameter in the presence of the drug molecule. Examples of the activity-inducible fusion proteins include chimeric antigen receptors (CAR) wherein the relevant physiological parameter is antigen binding.
Type:
Application
Filed:
February 11, 2022
Publication date:
April 25, 2024
Applicant:
Seattle Children's Hospital d/b/a Seattle Children's Research Institute
Inventors:
Ryan Koning, Adam Johnson, Chris Saxby, Michael C. Jensen, Ian Blumenthal, Aquene Reid
Abstract: The present disclosure provides improved compositions for the homology directed repair of the human globin locus for the prevention, treatment, or amelioration of at least one symptom of a hemoglobinopathy.
Type:
Application
Filed:
June 29, 2023
Publication date:
April 18, 2024
Applicants:
Seattle Children's Hospital (d/b/a Seattle Children's Research Institute), Fred Hutchinson Cancer Center
Inventors:
Andrew Scharenberg, Kyle Jacoby, Hans-Peter Kiem, David J. Rawlings, Christopher Lux, Sowmya Pattabhi, Olivier M. Humbert
Abstract: An intravascular ultrasound (IVUS) catheter-based device for targeted delivery of ultrasound-activated emulsions of oxygen-scavenging droplets into a bloodstream of patients suffering from a hypoxic condition and methods utilizing the device to protect the patients from reperfusion injury upon restoration of oxygenated blood to a hypoxic tissue.
Type:
Grant
Filed:
May 18, 2018
Date of Patent:
April 9, 2024
Assignees:
University of Cincinnati, Children's Hospital Medical Center
Inventors:
Kevin Haworth, Christy K. Holland, Karla Mercado-Shekhar, Andrew Redington, Bryan H. Goldstein
Abstract: A CD19-OR-CD20 chimeric antigen receptor (CAR) protein construct is provided. Also provided are nucleic acids encoding the CD19-OR-CD20 CAR; and methods of use, e.g. in the treatment of B cell malignancies. The CD19-OR-CD20 CAR of the invention is a bispecific CAR that can trigger T-cell activation upon detection of either CD19 or CD20 (or both). It is a single molecule that confers two-input recognition capability upon human T cells engineered to stably express this CAR.
Type:
Grant
Filed:
August 25, 2023
Date of Patent:
April 2, 2024
Assignees:
The Regents of the University of California, Seattle Children's Hospital
Inventors:
Yvonne Y. Chen, Eugenia Zah, Michael C. Jensen
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 30, 2023
Publication date:
March 28, 2024
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Abstract: The present disclosure relates to the co-expression of an endonuclease with an end-processing enzyme for the purpose of enhanced processing of the polynucleotide ends generated by endonuclease cleavage.
Type:
Application
Filed:
December 1, 2023
Publication date:
March 28, 2024
Applicant:
SEATTLE CHILDREN'S HOSPITAL (dba SEATTLE CHILDREN'S RESEARCH INSTITUTE)
Inventors:
Andrew M. SCHARENBERG, Michael T. CERTO, Kamila Sabina GWIAZDA
Abstract: Early detection of lysosomal storage diseases (LSDs) including Mucopolysaccharidosis Type I (MPS I) and Pompe Disease can greatly improve patient outcome as each disease can be fatal once symptoms emerge. Screening for MPS I and Pompe Disease using biological samples including dried blood spots (DBS), buccal swab, peripheral blood mononuclear cells (PBMCs), or white blood cells (WBCs) is described. The disclosed methods and assays provide a robust way to screen newborns for LSDs. The disclosed methods and assays can also allow rapid prediction of whether a patient with LSD will develop an immune response to enzyme replacement therapy (ERT), thus improving treatment for patients with LSDs. The disclosed methods and assays can also further reduce the number of false positives caused by pseudo deficiency cases of LSD, such as MPS I and Pompe Disease.
Type:
Grant
Filed:
March 31, 2021
Date of Patent:
March 26, 2024
Assignee:
Seattle Children's Hospital
Inventors:
Sihoun Hahn, Christopher Collins, Remwilyn Dayuha, Fan Yi
Abstract: Polynucleotides and vectors can be used for the expression of a transgene in cells, such as liver cells. The expression of the transgene from the polynucleotides and vectors can be useful in gene therapy. Various methods can be used for expressing the transgene from the polynucleotides and vectors in liver cells.
Type:
Grant
Filed:
January 10, 2018
Date of Patent:
March 26, 2024
Assignees:
THE SYDNEY CHILDREN'S HOSPITALS NETWORK (RANDWICK AND WESTMEAD (INCORPORATING THE ROYAL ALEXANDRA HOSPITAL FOR CHILDREN), CHILDREN'S MEDICAL RESEARCH INSTITUTE
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:
April 26, 2022
Date of Patent:
March 26, 2024
Assignee:
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Abstract: The present application relates to plasma cells and plasma cell precursors that express a macromolecule, such as a protein, protein mimetic or a peptide and compositions comprising these plasma cells or plasma cell precursors. The application further relates to methods of using and making the plasma cells and plasma cell precursors that express the macromolecule. Methods of treatment comprising administering the plasma cells or plasma cell precursors are also contemplated.
Type:
Grant
Filed:
March 14, 2018
Date of Patent:
March 26, 2024
Assignee:
Seattle Children's Hospital
Inventors:
David J. Rawlings, Richard James, Shaun W. Jackson, Iram Khan, King Hung, Andrew M. Scharenberg
Abstract: Aspects of the invention described herein relate to methods of making and using inducible promoters for transgene expression. The inducible promoters are derived from the NFAT-RE inducible system and are used to improve or enhance T cell survival and proliferation.
Type:
Application
Filed:
November 27, 2023
Publication date:
March 21, 2024
Applicant:
SEATTLE CHILDREN'S HOSPITAL (D/B/A SEATTLE CHILDREN'S RESEARCH INSTITUTE)
Abstract: An anti-ANGPTL3 antibody or an antigen-binding fragment thereof. The present invention provides an anti-ANGPTL3 antibody or an antigen-binding fragment thereof, wherein a heavy chain variable region includes complementarity determining regions (CDRs), including CDR-H1 having an amino acid sequence as set forth in SEQ ID No. 1, CDR-H2 having an amino acid sequence as set forth in SEQ ID No. 2, and CDR-H3 having an amino acid sequence as set forth in SEQ ID No. 3; and the CDRs of a light chain variable region includes CDR-L1 having an amino acid sequence as set forth in SEQ ID No. 4, CDR-L2 having an amino acid sequence as set forth in SEQ ID No. 5, and CDR-L3 having an amino acid sequence as set forth in SEQ ID No. 6. The antibody or the antigen-binding fragment thereof provided in the present invention can specifically recognize ANGPTL3.
Type:
Application
Filed:
March 30, 2021
Publication date:
March 21, 2024
Applicant:
CHILDREN'S HOSPITAL OF FUDAN UNIVERSITY
Inventors:
HONG XU, QIAN SHEN, JIA RAO, YIHUI ZHAI, LI SUN, HAIMEI LIU, QIANYING LV, XINLI HAN
Abstract: Some embodiments of the invention include a nucleic acid molecule comprising natural nucleotides, non-natural nucleotides, an LNA which comprises one or more RNA core molecules, or an RNA molecule which comprises more than one RNA core molecule. Some embodiments of the invention include a nucleic acid molecule comprising an RNA molecule which comprises more than one RNA core molecule. Other embodiments of the invention include a nucleic acid molecule comprising a DNA molecule encoding the RNA molecule (e.g., vector or viral vector). Other embodiments include compositions or pharmaceutical compositions that comprise the nucleic acid molecule. Some embodiments of the invention comprise reducing miR-143 in a cell. Other embodiments of the invention include methods to deliver a protein across the BBB. Other embodiments include methods for treating disease (e.g., LSD), neuronopathic disease, neurodegenerative disease, Hurler syndrome, or MPS I). Additional embodiments of the invention are also discussed herein.
Abstract: Disclosed herein are products, methods, and uses for treating, ameliorating, delaying the progression of, and/or preventing a muscular dystrophy or a cancer including, but not limited to, facioscapulohumeral muscular dystrophy (FSHD) or a cancer associated with DUX4 expression or overexpression. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of double homeobox 4 (DUX4). Even more particularly, the disclosure provides microRNA (miRNA) for inhibiting or downregulating the expression of DUX4 and methods of using said miRNA to inhibit or downregulate DUX4 expression in cells and/or in cells of a subject having a muscular dystrophy or a cancer including, but not limited to, FSHD or a cancer associated with DUX4 expression or overexpression.
Type:
Application
Filed:
February 3, 2022
Publication date:
March 21, 2024
Applicant:
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL