Abstract: Described herein are aminoquinoline and aminoacridine based hybrids, pharmaceutical compositions and medicaments that include such aminoquinoline and aminoacridine based hybrids, and methods of using such compounds for diagnosing and/or treating infections, neurodegerative diseases or disorders, inflammation, inflammation associated diseases and disorders, and/or diseases or disorders that are treatable with dopamine agonists such as the restless leg syndrome.
Type:
Application
Filed:
February 28, 2013
Publication date:
January 22, 2015
Applicant:
The McLean Hospital Corporation
Inventors:
Diwan S. Rawat, Sunny Manohar, Ummadisetty Chinna Rajesh, Deepak Kumar, Anuj Thakur, Mohit Tripathi, Panyala Linga Reddy, Shamseer Kulangara Kandi, Satyapavan Vardhineni, Kwang-Soo Kim, Chun-Hyung Kim
Abstract: A method of storing mammalian cells or tissue (e.g., liver cells or hepatocytes) for subsequent use comprises the steps of: (a) contacting the cells or tissue in vitro to a choleretic agent in an effective amount; (b) combining said cells or tissue with a cryopreservative; (c) freezing said cells or tissue, and then (d) storing said frozen cells or tissue in frozen form for subsequent use.
Type:
Application
Filed:
July 14, 2014
Publication date:
January 22, 2015
Inventors:
Robin N. Coger-Simmons, Laura Whritenour Schrum, Balasubramanian Karthik Kumar, Charles Lee
Abstract: A lipoaspirate collection device to aid in the collection and processing of human tissue and fluid obtained during liposuction for use in point-of-care cell therapy. The collection device includes a collection body and a collection cap. The collection cap may have a fluid port, a lipoaspirate port, a vacuum port, and a relief valve. Within the central cavity of the collection device, a cone shaped may be positioned such that the apex of the cone is positioned underneath the lipoaspirate inlet through which the lipoaspirate fluid and tissue are introduced.
Abstract: The present invention provides compositions comprising polytheylyene-dialkyloxypropyl conjugates (PEG-DAA), liposomes, SNALP, and SPLP comprising such compositions, and methods of using such compositions, liposomes, SNALP, and SPLP.
Type:
Grant
Filed:
August 6, 2010
Date of Patent:
January 20, 2015
Assignee:
Protiva Biotherapeutics, Inc.
Inventors:
James Heyes, Ian MacLachlan, Ellen Grace Ambegia
Abstract: This application relates to a method for differentiating pluripotent stem cells (PSCs) into vascular bed cells. Moreover this application relates to a method for differentiating human embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs) into vascular bed cells based on linked steps of chemically defined medium inductions.
Type:
Application
Filed:
December 9, 2013
Publication date:
January 15, 2015
Applicant:
HOFFMANN-LA ROCHE INC.
Inventors:
Klaus Christensen, Martin Graf, Roberto Iacone, Christoph Patsch, Eva Christina Thoma
Abstract: The present invention relates to a method for inducing cardiac differentiation of a pluripotent stem cell, which comprises the steps of (1) culturing a pluripotent stem cell in a medium containing one or more WNT signaling activators, and (2) culturing a cell produced in the step (1) in a medium containing one or more WNT signaling inhibitor.
Abstract: Improved methods of culturing embryos in media having amounts of lactate that have not previously been recognized as beneficial for embryo development. Also, compositions, devices and kits related to the same.
Abstract: It is described a conditioned medium (CM) obtainable by culturing, in a liquid culture medium, placental mesenchymal stem cells isolated from the placental tissue of pregnant women not affected by preeclampsia. The conditioned medium of the invention includes at least IL-6, IL-10 and MCP-1 proteins and it is effective for the therapeutic treatment of preeclampsia.
Abstract: A human immature endocrine cell population and methods for making an immature endocrine cell population are provided. Specifically, immature beta cells and methods for production of immature beta cells are described. Immature beta cells co-express INS and NKX6.1 and are uni-potent and thereby develop into mature beta cells when implanted in vivo. The mature beta cells in vivo are capable of producing insulin in response to glucose stimulation.
Abstract: Methods are described for the delivery of one or more small interfering RNAs (siRNAs) to a eukaryotic cell using a bacterium. Methods are also described for using this bacterium to regulate gene expression in eukaryotic cells using RNA interference, and methods for treating an inflammatory disease or disorder. The bacterium includes one or more siRNAs or one or more DNA molecules encoding one or more siRNAs. Vectors are also described for use with the bacteria of the invention for causing RNA interference in eukaryotic cells.
Abstract: A method of treating cancer through use of guanosine 3?,5?-cyclic monophosphate (cyclic GMP). Cyclic GMP decreases the number of human breast cancer and prostate adenocarcinoma as well as small-cell and squamous lung cells in culture by 30% (1 ?M), 84% (1 mM), 31% (1 ?M), and 30% (1 ?M), respectively. Cyclic GMP decreases DNA synthesis in human pancreatic, breast, and prostate adenocarcinomas as well as small-cell and squamous cell carcinomas of the lung at its 1 ?M concentration by 51%, 54%, 56%, 50% and 52%, respectively. Cyclic GMP when infused for one week decreases the tumor volume of human pancreatic adenocarcinomas in athymic mice 95% compared to untreated animals with human pancreatic adenocarcinomas.
Abstract: The present invention relates to substantially homogenous populations of human retinal progenitor cells having the following positive surface markers: SSEA4, CD73, PTK7 and PSA-NCAM. The invention also relates to method for preparing such substantially homogeneous cell populations from human tissue using cell sorting techniques.
Abstract: The present invention provides a method of more efficiently producing pancreas cells, particularly pancreatic hormone-producing cells, a method of stably producing pancreas cells in a large amount by more efficiently inducing differentiation of stem cells into pancreas cells, a medicament containing a pancreas cells and a screening method using the cells.
Abstract: A method of treating a lymphocyte mediated inflammation in a subject including administering a therapeutically effective amount of a cell delivery composition to the subject, the cell delivery composition of the application including an immunosuppressive cell and a plurality of targeting moieties that bind to endothelial cell adhesion molecules expressed by endothelial cells as a result of a lymphocyte mediated inflammatory response in the subject, the targeting moieties coated on and linked to the immunosuppressive cell and enhancing adherence of the immunosuppressive cell to an endothelial cell at a site of lymphocyte mediated inflammation when administered to the subject systemically, wherein the cell delivery composition, suppresses lymphocyte mediated inflammation in the subject.
Type:
Grant
Filed:
August 16, 2010
Date of Patent:
January 13, 2015
Assignee:
Case Western Reserve University
Inventors:
James E. Dennis, Thomas John Kean, Inkap Ko
Abstract: Provided herein are methods and compositions for the diagnosis, prognosis and treatment of a cancer associated disorders using the Fhit gene.
Abstract: The present invention provides constructs including a tubular biodegradable polyglycolic acid scaffold, wherein the scaffold may be coated with extracellular matrix proteins and substantially acellular. The constructs can be utilized as an arteriovenous graft, a coronary graft, a peripheral artery bypass conduit, or a urinary conduit. The present invention also provides methods of producing such constructs.
Type:
Application
Filed:
September 24, 2014
Publication date:
January 8, 2015
Inventors:
Shannon L.M. Dahl, Laura E. Niklason, Juliana Blum, Justin T. Strader, William E. Tente, Heather L. Prichard, Joseph J. Lundquist
Abstract: The present invention is directed generally to eukaryotic cells comprising single-celled organisms that are introduced into the eukaryotic cell through human intervention and which transfer to daughter cells of the eukaryotic cell, and methods of introducing such single-celled organisms into eukaryotic cells. The invention provides single-celled organisms that introduce a phenotype to eukaryotic cells that is maintained in daughter cells. The invention additionally provides eukaryotic cells containing magnetic bacteria. The invention further provides eukaryotic cells engineered with single-celled organisms to allow for multimodal observation of the eukaryotic cells. Each imaging method (or modality) allows the visualization of different aspects of anatomy and physiology, and combining these allows the imager to learn more about the subject being imaged.
Abstract: The features of the present invention relate to compounds, compositions and methods useful for modulating the expression of vascular endothelial growth factor (VEGF), such as by the mechanism of RNA interference (RNAi). The compounds and compositions include iRNA agents that can be unmodified or chemically-modified.
Type:
Application
Filed:
July 7, 2014
Publication date:
January 8, 2015
Inventors:
Antonin de Fougerolles, Maria Frank-Kamenetsky, Muthiah Manoharan, Kallanthottathil Rajeev, Philipp Hadwiger
Abstract: An isolated human brown adipose tissue stem cell line. In one embodiment, the isolated human brown adipose tissue stem cell line expresses the markers CD9, SSEA4, CD44, CD90, CD166, CD73, but not CD14, CD34, CD45 or STRO-1. In another embodiment, the isolated human brown adipose tissue stem cell line expresses the genes UCP1, PPARGC1A, NRF1, FOXC2, CREB1, SIRT3, and WNT5A (REFX). In still another embodiment, the isolated human brown adipose tissue stem cell line is capable of differentiating into osteoblasts, chondrocytes, and adipocytes.
Abstract: Provided herein is a method of treating Respiratory Syncytial Virus (RSV) infection in a cell. The method involves administering the guanosine-rich oligonucleotide (GRO) AS 1411 (5?-GGTGGTGGTGG TTGTGGTGGTGGTGG-3?—also known as GR026B and AGRO100) to the cell.
Type:
Application
Filed:
February 15, 2013
Publication date:
January 8, 2015
Applicant:
University of Toronto
Inventors:
Richard George Hegele, Peter Mastrangelo, Theo Moraes
Abstract: An aqueous cell culture medium composition includes an aqueous cell culture solution configured to support the culture of mammalian cells. The composition further includes a synthetic polymer conjugated to a polypeptide dissolved in the aqueous cell culture solution. The synthetic polymer conjugated to a polypeptide is configured to attach to the surface of a cell culture article under cell culture conditions. Incubation of the aqueous cell culture medium composition on a cell culture surface under cell culture conditions results is attachment to the surface of the synthetic polymer conjugated to the polypeptide.
Type:
Application
Filed:
January 31, 2013
Publication date:
January 8, 2015
Inventors:
Stephen Joseph Caracci, David Henry, Jessica Jo Kelley, Mark Alan Lewis, Yue Zhou
Abstract: The present invention relates to the field of stem cell biology, in particular the lineage specific differentiation of pluripotent or multipotent stem cells, which can include, but is not limited to, human embryonic stem cells (hESC) in addition to nonembryonic human induced pluripotent stem cells (hiPSC), somatic stem cells, stem cells from patients with a disease, or any other cell capable of lineage specific differentiation. Specifically described are methods to direct the lineage specific differentiation of hESC and/or hiPSC into floor plate midbrain progenitor cells and then further into large populations of midbrain fate FOXA2+LMX1A+TH+ dopamine (DA) neurons using novel culture conditions.
Abstract: A packaging cell line that complements recombinant adenoviruses based on serotypes from subgroup B, preferably adenovirus type 35. The cell line may be derived from primary, diploid human cells that are transformed by adenovirus E1 sequences either operatively linked on one DNA molecule or located on two separate DNA molecules, the sequences being operatively linked to regulatory sequences enabling transcription and translation of encoded proteins. Also disclosed is a cell line derived from PER.C6® that expresses functional Ad35 E1B sequences. The Ad35-E1B sequences are driven by the E1B promoter or a heterologous promoter and terminated by a heterologous poly-adenylation signal. The cell lines are useful for producing recombinant adenoviruses designed for gene therapy and vaccination, and can also be used for producing human recombinant therapeutic proteins such as human growth factors and human antibodies.
Type:
Application
Filed:
July 18, 2014
Publication date:
January 8, 2015
Inventors:
Ronald Vogels, Menzo Jans Emco Havenga, Majid Mehtali
Abstract: The present application concerns mutant proteins of the fusion protein (F protein) of the parainfluenza virus (PIV) which are currently indexed as type 5 PIV (PIV-5 or PIV5) and type 2 PIV (PIV-2 or PIV2). The present application concerns products deriving therefrom, such as: nucleic acids, vectors, cells, fusion inhibitors of the antibody, aptamer, interfering RNA type; myelomas, hybridomas; stem and progenitor cells. The present application also concerns mutant proteins and products derived therefrom for use in medical and biotechnological applications.
Type:
Grant
Filed:
November 17, 2009
Date of Patent:
January 6, 2015
Assignees:
Centre National de la Recherche Scientifique, Universite Claude Bernard de Lyon 1, Les Hospices Civils de Lyon
Inventors:
Manuel Melchior Jean-Pierre Rosa Calatrava, Olivier Terrier, Francois Edouard Julien Durupt
Abstract: The present invention relates to compounds and compositions for expanding the number of CD34+ cells for transplantation. The invention further relates to a cell population comprising expanded hematopoietic stem cells (HSCs) and its use in autologous or allogeneic transplantation for the treatment of patients with inherited immunodeficient and autoimmune diseases and diverse hematopoietic disorders to reconstitute the hematopoietic cell lineages and immune system defense.
Type:
Grant
Filed:
October 29, 2009
Date of Patent:
January 6, 2015
Assignees:
IRM LLC, The Scripps Research Institute
Inventors:
Anthony E. Boitano, Michael Cooke, Shifeng Pan, Peter G. Schultz, John Tellew, Yongqin Wan, Xing Wang
Abstract: The invention relates to culture systems, methods, and conditions that allow pluripotent undifferentiated hESCs or iPSCs to progressively and uniformly differentiate into cells of the chondrogenic lineage.
Abstract: The present invention relates to a simplified process, which is shorter in time, for propagation of proliferating cells, such as e.g. progenitor or stem cells, by means of a biphasic culturing system having a differentiation supporting component and a proliferation supporting component, and to the use of the stem cell cultures obtained in this way for cell therapy purposes. The present invention invention describes a method, which is highly efficient to prime stem or progenitor cells to differentiation using non-attachment matrices and differentiation supporting component. The cells produced therefrom may be used to treat a variety of neurodegenerative disorders.
Abstract: A method of generating pancreatic progenitor cells is disclosed. The method comprises: (a) differentiating stem cells under conditions such that at least a portion of the cells express glucose transporter 2 (GLUT2) so as to generate GLUT2-expressing cells; and (b) enriching for the GLUT2-expressing cells so as to generate a population of GLUT2 enriched cells, wherein at least 80% of the population of GLUT2 enriched cells express GLUT2, thereby generating pancreatic progenitor cells. Isolated populations of cells generated according to the method, pharmaceutical compositions comprising same and uses thereof are also disclosed.
Type:
Grant
Filed:
April 12, 2011
Date of Patent:
January 6, 2015
Assignee:
Technion Research & Development Foundation Limited
Inventors:
Joseph Itskovitz-Eldor, Bettina Fishman, Hanna Segev
Abstract: Certain embodiments disclosed herein are directed to a method of producing endoderm cells, such as definitive endoderm cells by exposing stem cells such as embryonic stem cells or induced pluripotent stem (iPS) cells to an effective amount of at least one compound described herein to differentiate the stem cells into the endoderm cells such as definitive endoderm cells. Differentiated endoderm cells produced by the methods disclosed herein can be differentiated into pancreatic epithelium, and other endoderm derivatives such as thymus, liver, stomach, intestine and lung. Another aspect of the present invention relates to a method of producing pancreatic progenitor cells, such as Pdx1-positive pancreatic progenitor cells by exposing endoderm cells, such as definitive endoderm cells to an effective amount of at least one compound described herein to differentiate the definitive endoderm cells into Pdx1-positive pancreatic progenitor cells.
Type:
Grant
Filed:
August 12, 2013
Date of Patent:
January 6, 2015
Assignee:
President and Fellows of Harvard College
Inventors:
Douglas A. Melton, Malgorzata Borowiak, Rene Maehr, Shuibing C. Chen, Weiping Tang, Julia L. Fox, Stuart L. Schreiber
Abstract: The present invention is based on the discovery that a single lentiviral vector expressing multiple individual transcription factor proteins from a single multi-cistronic mRNA can reprogram a fibroblast cell to a stem cell-like cell. These reprogrammed induced pluripotent stem (iPS) cells are pluripotent. Additions of the Cre-LoxP sequences into the single lentiviral vector facilitate excision of the vector after reprogramming in achieved. Addition of a maker gene into the single lentiviral vector facilitates detection of the presence of the vector in an iPS. The invention provides compositions and methods of producing iPS cells using a single multi-cistronic lentiviral vector.
Abstract: Disclosed are methods of gene delivery using capsid-modified recombinant adeno-associated viral (rAAV) vectors. Exemplary methods are provided employing vectors that have altered affinity for heparin or heparin sulfate, as well as vectors, expression systems, and rAAV virions that lack functional VP2 protein expression, but are nevertheless, fully virulent. Also provided by the invention are methods employing the rAAV vector-based compositions, virus particles, host cells, and pharmaceutical formulations in the expression of selected therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in selected mammals, including organs, tissues, and human host cells.
Type:
Application
Filed:
July 8, 2014
Publication date:
January 1, 2015
Inventors:
Nicholas Muzyczka, Shaun R. Opie, Kenneth H. Warrington
Abstract: The invention describes novel virus-like particles for use as vaccines, diagnostic tools and R&D tools based on recombinant DNA and cell cultivation techniques for production. The recombinant virus-like particles of the invention are assembled by polypeptide chains that incorporate several, in particular two or more, different epitopes which are selected either (a) from different viral strains of the same virus and/or (b) from different serotypes of the same virus and/or (c) from different viral strains specific for different hosts. These epitopes are then displayed on the particle surface.
Type:
Application
Filed:
August 29, 2014
Publication date:
January 1, 2015
Inventors:
Corinne JOHN, Christian SCHAUB, Sabine WELLNITZ
Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.
Type:
Application
Filed:
February 8, 2013
Publication date:
January 1, 2015
Inventors:
Hongyan Qi, Soongyu Choi, Amal Dakka, Gary Mitchell Karp, Jana Narasimhan, Nikolai Naryshkin, Anthony A. Turpoff, Marla L. Weetall, Ellen Welch, Matthew G. Woll, Tianle Yang, Nanjing Zhang, Xiaoyan Zhang, Xin Zhao, Luke Green, Emmanuel Pinard, Hasane Ratni
Abstract: The present invention relates to certain novel shRNA molecules and methods of use thereof. According to certain embodiments of the present invention, methods for reducing the expression level of a target gene are provided. Such methods generally comprise providing a cell with one or more precursor nucleic acid sequences that encode two or more RNA molecules. A first RNA molecule comprises a double stranded sequence, which includes a guide strand sequence that is complementary to a portion of an mRNA transcript encoded by the target gene. In addition, a second RNA molecule comprises a second double stranded sequence, which includes a second guide strand sequence that is partially complementary to a portion of the mRNA transcript encoded by the target gene. Preferably, the second guide strand sequence comprises one or more bases that are mismatched with a nucleic acid sequence of the mRNA transcript encoded by the target gene.
Abstract: Mesenchymal stem cells which express TNF-? receptor Type I in an amount of at least 13 pg/106 cells. Such mesenchymal stem cells inhibit the proliferation of lymphocytes and may be employed, in particular, in the treatment of graft-versus-host disease.
Type:
Application
Filed:
September 12, 2014
Publication date:
January 1, 2015
Inventors:
Alla Danilkovitch, Diane Carter, Alicia Tyrell, Simon Bubnic, Michelle Marcelino, Rodney Monroy
Abstract: The subject matter of the present invention is a device for guiding cell migration comprising a substrate having a textured surface intended to be brought into contact with cells, said textured surface having an anisotropic three-dimensional structure consisting of a network of projections inclined relative to the normal to the plane formed by said textured structure, in the direction imparted by said anisotropic structure. The invention also concerns, according to another aspect, a method for guiding cell migration including the bringing into contact of cells with a substrate having a textured surface and an anisotropic three-dimensional structure, said structure consisting of projections inclined as previously described. The device or method according to the invention can in particular be applied in the fields of dermatology, implantology and tissue engineering.
Type:
Application
Filed:
September 17, 2012
Publication date:
January 1, 2015
Inventors:
Mael Le Berre, Matthieu Piel, Yong Chen, Yanjun Liu
Abstract: The technology described herein is directed to methods and compositions relating to the differentiation and activity of brown adipocytes, and the therapeutic uses thereof.
Type:
Application
Filed:
November 28, 2012
Publication date:
January 1, 2015
Applicant:
THE GENERAL HOSPITAL CORPORATION
Inventors:
Chad Cowan, Robert Schinzel, Tim Ahfeldt, YounKyoung Lee
Abstract: Provided are valencene synthase polypeptides, nucleic acid molecules encoding the valencene synthases, host cells containing the nucleic acids and methods for producing products whose production is catalyzed by the polypeptides. Also provided are methods for producing valencene and nootkatone.
Abstract: The invention relates to compositions of vault complexes containing recombinant cytokine fusion proteins that include a cytokine and a vault targeting domain, and methods of using the vault complexes to deliver the cytokines to a cell or subject, and methods for using the compositions to treat cancer, such as lung cancer.
Type:
Grant
Filed:
November 2, 2010
Date of Patent:
December 30, 2014
Assignees:
The Regents of the University of California, The United States of America Represented by the Department of Veterans Affairs
Inventors:
Leonard H. Rome, Valerie A. Kickhoefer, Steven M. Dubinett, Sherven Sharma, Upendra K. Kar
Abstract: The invention relates to therapeutic agents for use in the prevention or treatment of Alzheimer's disease. In particular the invention relates to use of inhibitors of cell cycle reentry and progression to the G1/S transition or inhibitors of progression of the cell cycle through the G1/S transition point in the prevention or treatment of Alzheimer's disease.
Abstract: Compositions and methods are provided for user-specified fine-tuned protein expression levels, by controlling the initiation of protein translation; and a model for analysis of expression. This method of control can be used to vary, tune, and optimize protein production in genetically engineered organisms, cells, or devices.
Type:
Application
Filed:
January 11, 2013
Publication date:
December 25, 2014
Inventors:
Clifford Lee Wang, Joshua Paul Ferreira
Abstract: The present invention relates to methods of optimization of a protein coding sequences for expression in a given host cell. The methods apply genetic algorithms to optimise single codon fitness and/or codon pair fitness sequences coding for a predetermined amino acid sequence. In the algorithm generation of new sequence variants and subsequent selection of fitter variants is reiterated until the variant coding sequences reach a minimum value for single codon fitness and/or codon pair fitness. The invention also relates to a computer comprising a processor and memory, the processor being arranged to read from and write into the memory, the memory comprising data and instructions arranged to provide the processor with the capacity to perform the genetic algorithms for optimisation of single codon fitness and/or codon pair fitness.
Type:
Application
Filed:
May 20, 2014
Publication date:
December 25, 2014
Inventors:
Johannes Andries ROUBOS, Noel Nicolaas Maria Elisabeth VAN PEIJ
Abstract: The invention relates to induction of reprogramming of somatic cells, by methods which require mild growth conditions. Disclosed are methods of inducing dedifferentiation of mesenchymal stromal cell (MSC), by seeding or incubating mesenchymal stromal cells (MSCs) at low density, and without introduction or expression of exogenous genes in the cells.
Abstract: The present invention relates to genetically modified cells that are capable of optimal transgene expression by co-expressing a silencing suppressor whilst at the same time are also capable of silencing a gene, such as a naturally occurring gene of the cell. The present invention also relates to methods of producing the modified cells, as well as relates to processes for obtaining a genetically modified cell with a desired property.
Type:
Application
Filed:
December 21, 2012
Publication date:
December 25, 2014
Applicants:
Commonwealth Scientific and Industrial Research Organisation, Grains Research and Development Corporation
Inventors:
Craig Christopher Wood, Fatima Naim, Surinder Pal Singh
Abstract: The present invention relates to a cell for producing a secreted protein comprising a polynucleotide comprising a nucleic acid sequence encoding a fast cycling cdc42 mutant and a polynucleotide comprising a nucleic acid sequence encoding a secreted protein. It also relates to a method for producing said cell and to a method for producing a secreted protein using said cell.
Type:
Application
Filed:
May 14, 2012
Publication date:
December 25, 2014
Applicant:
PROBIOGEN AG
Inventors:
Volker Sandig, Karsten Winkler, Henning Von Horsten, Thomas Rose
Abstract: The invention provides an isolated H3 equine influenza A virus, as well as methods of preparing and using the virus, and genes or proteins thereof.
Type:
Application
Filed:
April 17, 2014
Publication date:
December 25, 2014
Applicant:
Wisconsin Alumni Research Foundation
Inventors:
Christopher W. Olsen, Gabriele A. Landolt, Alexander I. Karasin
Abstract: The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.
Abstract: Articles and methods for stem cell differentiation are generally described. In some embodiments, an article for stem cell differentiation may comprise an oxygen permeable substrate having at least a portion of a surface coated with a matrix. The matrix may allow the surface chemistry of the substrate to be altered, such that the cell-substrate surface interactions may be finely controlled without substantially affecting the oxygen permeability of the substrate. The surface chemistry may be altered to promote directed stem cell differentiation by, e.g., modification of the matrix surface with a specific density of biological molecules. In some embodiments, methods for stem cell differentiation may comprise directing the differentiation of stem cells on the articles, described herein, under suitable environmental conditions.
Type:
Application
Filed:
June 13, 2014
Publication date:
December 18, 2014
Applicant:
Massachusetts Institute of Technology
Inventors:
Clark K. Colton, Karen K. Gleason, Anna M. Coclite, Amanda R. Diienno