Abstract: The invention provides compositions and methods for allele specific gene editing. In particular, the invention provides methods and compositions for treating dominant progressive hearing loss by selectively inactivating a dominant mutation in TMC1.
Type:
Application
Filed:
December 20, 2021
Publication date:
April 14, 2022
Applicants:
Children's Medical Center Corporation, President and Fellows of Harvard College
Abstract: Antibodies, and antigen-binding fragments thereof, that specifically bind to Cluster of Differentiation 1a (CD1a) are provided. Embodiments include uses, and associated methods of using the antibodies, and antigen-binding fragments thereof.
Type:
Application
Filed:
October 7, 2021
Publication date:
April 14, 2022
Applicants:
Pfizer Inc., Children's Medical Center Corporation
Inventors:
Florian WINAU, Oleg V. KOVALENKO, Chew Shun CHANG, Di WU, Nicholas Andrew MARZE, Shian-Huey CHIANG
Abstract: The present invention is based, in part, on the identification of novel methods for defining predictive biomarkers of response to anti-cancer drugs.
Type:
Grant
Filed:
December 28, 2018
Date of Patent:
April 12, 2022
Assignees:
Children's Medical Center Corporation, The Technical University of Denmark, Dana-Farber Cancer Institute, Inc., The Brigham and Women's Hospital, Inc.
Inventors:
Zoltan Szallasi, Nicolai Juul Birkbak, Aron Eklund, Daniel Silver, Zhigang Wang, Andrea Richardson
Abstract: Embodiments herein disclose methods relating to diabetic nephropathy (DN); methods for preventing the onset and also for preventing the progressing of DN, as well as the treatment of DN in diabetic subjects comprising administering reparixin and/or ladarixin which are inhibitors of CXCL8 receptor CXCR1 and CXCR2 activation.
Type:
Grant
Filed:
October 3, 2017
Date of Patent:
April 5, 2022
Assignees:
THE CHILDREN'S MEDICAL CENTER CORPORATION, DOMPE FARMACEUTICI S.P.A.
Inventors:
Paolo Fiorina, Roberto Bassi, Andrea Vergani, Marcello Allegretti
Abstract: The invention provides compositions comprising mesenchymal stem cell (MSC) derived exosomes, and methods of their use in subjects having certain lung diseases including inflammatory lung disease.
Type:
Application
Filed:
October 1, 2021
Publication date:
March 31, 2022
Applicant:
Children's Medical Center Corporation
Inventors:
S. Alexander Mitsialis, Changjin Lee, Stella Kourembanas
Abstract: Methods for preparing and using collagen extracts and collagen scaffolds are provided. Additionally, methods and related kits for the repair of articular tissue using the collagen material are provided.
Type:
Application
Filed:
November 4, 2021
Publication date:
March 31, 2022
Applicant:
The Children's Medical Center Corporation
Abstract: Provided herein are Clostridial Botulinum neurotoxin (BoNT) polypeptides of a novel serotype (BoNT/X) and methods of making and using the BoNT polypeptides, e.g., in therapeutic applications.
Abstract: In at least some aspects, an instrument port for introducing an instrument into a surgical site comprises: a port body having: an instrument channel extending through the port body; and a plurality of fluid flush channels each separate from one another and the instrument channel, each extending along a major portion of the port body and in fluid communication with the instrument channel; and a bulb comprising a bulb channel extending through the bulb, the bulb channel aligned with the instrument channel, wherein the bulb channel and instrument channel are configured to receive the instrument.
Type:
Grant
Filed:
March 9, 2018
Date of Patent:
March 29, 2022
Assignee:
The Children's Medical Center Corporation
Abstract: The present invention provides for methods of epigenetic analysis. In some cases, the methods may include obtaining a sample comprising a nucleic acid sequence. In some cases, the nucleic acid sequence may comprise one or more epigenetic marks. The methods may include performing a sequencing. The methods may include distinguishing a hydroxymethylated base from a methylated base.
Type:
Application
Filed:
November 17, 2021
Publication date:
March 24, 2022
Applicants:
The Children's Medical Center Corporation, The United States of America, As Represented by the Secretary, Department of Health & Human Services
Abstract: Covalently modified alginate polymers, possessing enhanced biocompatibility and tailored physiochemical properties, as well as methods of making and use thereof, are disclosed herein. The covalently modified alginates are useful as a matrix for coating of any material where reduced fibrosis is desired, such as encapsulated cells for transplantation and medical devices implanted or used in the body.
Type:
Grant
Filed:
September 26, 2019
Date of Patent:
March 8, 2022
Assignees:
MASSACHUSETTS INSTITUTE OF TECHNOLOGY, THE CHILDREN'S MEDICAL CENTER CORPORATION
Inventors:
Arturo J. Vegas, Joshua C. Doloff, Omid Veiseh, Minglin Ma, Robert S. Langer, Daniel G. Anderson
Abstract: Described herein are modified integrin ? and/or ? headpiece polypeptides, and crystallizable integrin polypeptide dimers comprising a modified integrin ? and/or ? headpiece polypeptide and a disulfide bond linking the two integrin headpiece polypeptide subunits. Methods for using the modified integrin ? and/or ? headpiece polypeptides and the integrin polypeptide dimers are also provided herein. For example, methods for characterizing integrin-ligand interaction and identifying integrin ligands are also provided herein. In some embodiments, the identified integrin ligands can be used as inhibitors of integrins.
Type:
Application
Filed:
July 23, 2021
Publication date:
March 3, 2022
Applicant:
THE CHILDREN'S MEDICAL CENTER CORPORATION
Inventors:
Timothy Alan SPRINGER, Xianchi Dong, Chafen Lu
Abstract: Embodiments herein relate to in vitro production methods of hematopoietic stem cell (HSC) and hematopoietic stem and progenitor cell (HSPC) that have long-term multilineage hematopoiesis potentials upon in vivo engraftment. The HSC and HSPCs are derived from pluripotent stem cells-derived hemogenic endothelia cells (HE).
Type:
Grant
Filed:
May 3, 2017
Date of Patent:
March 1, 2022
Assignee:
THE CHILDREN'S MEDICAL CENTER CORPORATION
Abstract: The disclosure provides liposomes (e.g., cancer-targeting liposomes) with ligands (e.g., EGFR ligands and ICAM-1 ligands) conjugated to liposome surfaces. In some embodiments, the molecular ratio of different ligands complement the relative molecular density (i.e., ratio) of overexpressed protein on the surface of a cell targeted by the liposome (e.g., cancer cell).
Type:
Grant
Filed:
March 16, 2018
Date of Patent:
March 1, 2022
Assignee:
Children's Medical Center Corporation
Inventors:
Marsha A. Moses, Peng Guo, Jiang Yang, Debra Auguste, Daxing Liu
Abstract: The present disclosure provides compounds of Formula (I). The compounds described herein may be useful in treating and/or preventing proliferative diseases (e.g., cancer). Also provided in the present disclosure are pharmaceutical compositions, kits, methods, and uses including or using a compound described herein.
Type:
Application
Filed:
May 4, 2021
Publication date:
February 24, 2022
Applicants:
Children's Medical Center Corporation, Beth Israel Deaconess Medical Center, Inc.
Abstract: Embodiments herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels.
Type:
Application
Filed:
August 17, 2021
Publication date:
February 24, 2022
Applicant:
THE CHILDREN'S MEDICAL CENTER CORPORATION
Inventors:
Michael MILSOM, David A. WILLIAMS, Richard GREGORY
Abstract: Provided herein are Clostridial Botulinum neurotoxin (BoNT) polypeptides of a novel serotype (BoNT/X) and methods of making and using the BoNT polypeptides, e.g., in therapeutic applications.
Abstract: The present invention provides compositions and methods for delivery of therapeutic agents across an barrier. The compositions include a therapeutic agent (e.g., antimicrobial agent, antibiotic, or anesthetic agent), a permeation enhancer which increases the flux of the therapeutic agent across the barrier, and a matrix forming agent. The matrix forming agent forms a gel at a suitable gelation temperature and rheological properties for use in drug delivery, and in some cases, the gelation temperature and rheological properties are not significantly changed from those of the composition without the permeation enhancer. The invention also provides a matrix forming agent and compositions thereof. Such compositions are particularly useful in the treatment of otitis media. Methods of treatment, methods of delivery, and kits for the compositions described herein are also provided.
Type:
Application
Filed:
July 23, 2021
Publication date:
February 10, 2022
Applicants:
Children's Medical Center Corporation, Massachusetts Institute of Technology
Inventors:
Daniel S. Kohane, Rong Yang, Lily Yun Lin
Abstract: The present invention provides systems and methods for identifying, isolating, and/or characterizing microRNAs, their targets, and microRNA response elements, and for predicting their biological function.