Abstract: The present invention relates to novel therapeutic ways for treating Wolfram Syndrome (WS) by targeting the neuronal calcium sensor 1 (NCS1). The present inventors have demonstrated that WFS1, which loss of function is responsible of the Wolfram Syndrome, forms a complex with the neuronal calcium sensor 1 (NCS1). The inventors have further demonstrated that WFS1 associates with NCS1 to prevent its degradation by the proteasome and that NCS1 regulates VDAC expression and mitochondrial respiratory chain. Thus, present invention provides an agonist of NCS1 for use in the treatment of WS. Such an agonist is e.g. a NCS1-encoding polynucleotide, an inhibitor of the proteasome or of calpains. The inventors have further shown that overexpression of NCS1 in WS cells allows increasing complex II driven respiration. The present invention further relates to a method for predicting the severity of WS by measuring the NCS1 level in a sample obtained from a patient.
Type:
Grant
Filed:
March 23, 2017
Date of Patent:
May 5, 2020
Assignees:
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), UNIVERSITE DE MONTPELLIER
Inventors:
Benjamin Delprat, Cécile Cribaillet-Delettre, Claire Angebault
Abstract: The present invention relates to compositions and methods for creation of vector nucleic acid sequences (e.g., retroviral nucleic acid sequences) that comprise two or more exogenous nucleic acid sequences that encode highly homologous (e.g., identical) polypeptide sequences, yet wherein at least one of the exogenous nucleic acid sequences has been mutated using degenerate codons for purpose of reducing homology between the two or more exogenous nucleic acid sequences while maintaining the encoded polypeptide sequence. Preferred nucleic acid sequences include those encoding multi-chimeric immune receptor (CIR) genes. Specific nucleic acid sequences of such CIR genes are also disclosed.
Type:
Grant
Filed:
December 4, 2015
Date of Patent:
February 4, 2020
Assignee:
ROGER WILLIAMS MEDICAL CENTER
Inventors:
Wen Yang, Richard P. Junghans, Anthony J. Bais
Abstract: The present invention refers to a device, comprising a hollow body having at least one open end comprising at least one solid matrix binding, adsorbing, absorbing, chelating or retaining compounds which are not desired in a sample and preferably at least one barrier which is non-permeable for liquids and solids under ambience conditions, however, becomes liquid-permeable by applying an external force to the barrier, the use of such a device for isolating or purifying a biomolecule from a sample, a method for preparation of the device and a method for isolation or purification of any biomolecule using said device.
Type:
Grant
Filed:
July 13, 2011
Date of Patent:
December 24, 2019
Assignee:
QIAGEN GmbH
Inventors:
Vera Holländer, Markus Müller, Karin Schulte
Abstract: The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
Type:
Grant
Filed:
August 23, 2016
Date of Patent:
December 3, 2019
Assignee:
The Regents of the University of California
Inventors:
David V. Schaffer, Ryan R. Klimczak, James T. Koerber, John G. Flannery
Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.
Abstract: Provided herein are neuroprotective molecules containing a sequence of 25-35 contiguous nucleotides between nucleotide 1 and nucleotide 50 of a mature human tRNA and at least four contiguous guanosine-containing nucleotides, where the sequence of 25-35 contiguous nucleotides contains a D-loop stem structure, the at least four contiguous guanosine-containing nucleotides are located at the 5? end of the neuroprotective molecule, and the neuroprotective molecule contains at least one deoxyribonucleotide. Also provided are neuroprotective molecules containing a sequence of 25-35 contiguous between nucleotide 1 and nucleotide 50 of a mature human tRNACYS; and at least four contiguous guanosine-containing nucleotides, where the sequence of 25-35 contiguous nucleotides contains a D-loop stem structure and the at least four contiguous guanosine-containing nucleotides are located at the 5? end of the neuroprotective molecule.
Type:
Grant
Filed:
May 9, 2017
Date of Patent:
October 29, 2019
Assignee:
The Brigham and Women's Hospital, Inc.
Inventors:
Paul Anderson, Pavel Ivanov, Mohammed Emara
Abstract: The present invention relates to a viral vector for expression of the cell death-inducing PUMA protein, for use in gene therapy, for example for the treatment of rheumatoid arthritis. This vector consists in particular of a recombinant adenovirus expressing the gene encoding the PUMA protein and of a recombinant baculovirus containing a coxsackie-adenovirus receptor (CAR).
Type:
Grant
Filed:
October 7, 2014
Date of Patent:
October 22, 2019
Assignees:
Hospices Civils de Lyon, The Regents of the University of California, Universite Claude-Bernard-Lyon 1, Institut D'Enseignement Superieur et de Recherche en Alimentation, Sante Animale, Sciences Argonomiques et de l'Environnement, Institut National de la Sante et de la Recherche Medicale
Inventors:
Pierre Miossec, Saw-See Hong, Gary Firestein
Abstract: Provided are methods, systems, and kits for cell processing, e.g., for therapeutic use, such as for adoptive cell therapy. The provided methods include transduction methods, in which cells and virus are incubated under conditions that result in transduction of the cells with a viral vector. The incubation in some embodiments is carried out in an internal cavity of a generally rigid centrifugal chamber, such as a cylindrical chamber made of hard plastic, the cavity of which may have a variable volume. The methods include other processing steps, including those carried out in such a chamber, including washing, selection, isolation, culture, and formulation. In particular, the disclosure relates to method providing advantages over available processing methods, such as available methods for large-scale processing. Such advantages include, for example, reduced cost, streamlining, increased efficacy, increased safety, and increased reproducibility among different subjects and conditions.
Type:
Grant
Filed:
November 4, 2015
Date of Patent:
October 1, 2019
Assignee:
Juno Therapeutics, Inc.
Inventors:
Ryan L. Crisman, Chris Ramsborg, Travis Wood
Abstract: Some embodiments of the invention comprise methods, systems, and compositions to selectively induce, whether in vitro or in vivo, the neuronal differentiation of multipotent stromal cells through the application of microRNAs, including but not limited to miRNA-124, miRNA-137 and/or miRNA-9* expression products of those miRNAs, and molecules and compositions containing functional elements of those miRNAs. Some embodiments of the invention also comprise the therapeutic administration and use of such induced cells to treat mammalian injuries and diseases, including but not limited to, nervous system injuries or diseases that may otherwise result in decreased cell or system function.
Abstract: Methods for increasing specificity of RNA-guided genome editing, e.g., editing using CRISPR/Cas9 systems, using truncated guide RNAs (tru-gRNAs).
Type:
Grant
Filed:
February 10, 2017
Date of Patent:
September 17, 2019
Assignee:
The General Hospital Corporation
Inventors:
J. Keith Joung, Jeffry D. Sander, Morgan Maeder, Yanfang Fu
Abstract: Correlations between polymorphisms and breast cancer are provided. Methods of diagnosing, prognosing, and treating breast cancer are provided. Systems and kits for diagnosis, prognosis and treatment of breast cancer are provided. Methods of identifying breast cancer modulators are also described.
Type:
Grant
Filed:
June 7, 2017
Date of Patent:
September 10, 2019
Assignee:
Cambridge Enterprise Limited
Inventors:
David Cox, Dennis Ballinger, Bruce Ponder, Doug Easton
Abstract: The present invention relates to a method for generating batches of lymphocytes with averaged potency. In particular, the present invention relates to a method of pooling lymphocytes from different donors to avoid NK alloreactivity and anti-HLA immune response. Lymphocytes from each donor are inactivated for at least a gene encoding a TCR component, and are pooled together before be administrated to a subject in need thereof. Thus, this method allows generating batches of lymphocytes with averaged potency, particularly to treat cancer, viral infection or auto-immune disease. The present invention also relates to a batch of lymphocytes obtainable by this method. The batch of lymphocytes can be used to be administrated to one or several patients, being made available as an “off the shelf” therapeutic product, in particular to treat cancer, auto-immune disease or viral infection.
Type:
Grant
Filed:
November 21, 2014
Date of Patent:
July 23, 2019
Assignee:
CELLECTIS
Inventors:
David Sourdive, Carole Desseaux, Andrew Scharenberg
Abstract: The subject matter of the present invention is in particular the use of an amino acid sequence of IDE, or of an analogue or fragment thereof, or of at least one nucleic acid sequence encoding this sequence, as a biomarker, or as an active agent, with regard to a dandruff condition of the scalp.
Type:
Grant
Filed:
December 12, 2011
Date of Patent:
July 16, 2019
Assignee:
L'OREAL
Inventors:
Caroline Delattre, Philemon Sirven, Dominique Bernard
Abstract: The present disclosure provides a controlled release composition comprising a plurality of microparticles and a matrix, wherein: the plurality of microparticles comprises a first material; the matrix comprises a second material; and the melting temperature of the first material is higher than the melting temperature of the second material. Also provided are methods of making and using the same.