Abstract: Compositions and methods for treatment of mitochondrial respiratory chain dysfunction and other mitochondrial disorders are provided. Also disclosed are a number of screening assays having utility for the identification of agents which modulate the phenotype associated with mitochondrial respiratory chain dysfunction.
Type:
Application
Filed:
November 17, 2023
Publication date:
June 6, 2024
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Abstract: Disclosed herein are systems and methods for respiration-controlled virtual experiences. In an embodiment, a controller presents a virtual experience via a user interface that is perceptible to a user. The controller receives a respiration-data stream from a respiration device with which the user is operably engaged. The respiration-data stream includes one or more respiration-parameter values of one or more respiration parameters associated with ongoing respiration of the user. The controller updates the virtual experience based at least in part on the one or more respiration-parameter values in the respiration-data stream. The virtual experience includes a plurality of sequential phases that have an associated phase sequence. Among the sequential phases is an active-induction phase that corresponds in time to the user receiving anesthetic induction via the respiration device, as well as one or more phases that each precede the active-induction phase.
Abstract: Provided are chimeric VEGF-binding proteins and nucleic acids (e.g., a vector) encoding chimeric VEGF-binding proteins, methods and host cells for producing these proteins and nucleic acids, and pharmaceutical compositions containing these proteins and nucleic acids. Also provided are methods of treating an angiogenic disease or disorder that include administering at least one of the chimeric VEGF-binding proteins or at least one of the nucleic acids (e.g., a vector) encoding a chimeric VEGF-binding protein.
Type:
Grant
Filed:
March 4, 2019
Date of Patent:
June 4, 2024
Assignees:
Children's Medical Center Corporation, The General Hospital Corporation, President and Fellows of Harvard College
Inventors:
Bob Carter, Jeng-Shin Lee, Szofia S. Bullain, Richard C. Mulligan
Abstract: A frequency encoded source imaging system includes an EEG or MEG sensor array and a processing system for analyzing the signals from the sensor array in at least two different frequency bands, where the analysis is localized with respect to a three-dimensional grid corresponding to the portion of the human body.
Abstract: The present application relates to, inter alia, compositions including proteins for expression in host cells to render them resistant to rapamycin. The application further relates to methods of using the proteins, cells, and compositions disclosed therein for modulating cell signaling and for selective expansion of cells.
Type:
Grant
Filed:
April 25, 2019
Date of Patent:
May 21, 2024
Assignee:
Seattle Children's Hospital
Inventors:
Andrew M. Scharenberg, David J. Rawlings, Karen Sommer, Samuel West, Yuchi Chiang Honaker, Ryo Takeuchi
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: 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: 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: 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: 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