Abstract: The disclosure provides, among other things, the use of GLP-1 receptor agonist compounds to treat obstructive sleep apnea. The GLP-1 receptor agonist compounds may be exendins, exendin analogs, GLP-1 (7-37), GLP-1 (7-37) analogs (e.g., GLP-1 (7-36)-NH2) and the like. The GLP-1 receptor agonist compound may be exenatide.
Type:
Application
Filed:
August 26, 2016
Publication date:
December 15, 2016
Applicants:
Amylin Pharmaceuticals, LLC, ELI LILLY & COMPANY
Abstract: The present disclosure provides methods and uses for treating lymphocyte related cancers comprising administering a nutrient stress-inducing agent in combination with an agent that lowers the levels of or inhibits TRAF1. Also provided are methods of determining treatment for a subject with a lymphocyte related cancer and assays for identifying a PKN1 inhibitor.
Type:
Application
Filed:
March 6, 2015
Publication date:
December 15, 2016
Inventors:
Ali Abdul Sater, Ann Josephine McPherson, Tania Helen Watts, Mark Minden, Rob Laister
Abstract: Viral infections of the eye, and particularly viral infections in the Herpesviridae and Adenoviridae families, can be treated by administration of a pharmaceutical made up of an enzymatically active ribonuclease and a vehicle. Advantageously, the enzymatically active ribonuclease is ranpirnase, the '805 variant, rAmphinase 2, and Amphinase 2, and the vehicle is an aqueous solution.
Abstract: A topical sting/bite treatment compound and method of making same comprising combining effective amounts of: a natural enzyme; a natural abrasive; a natural pain reliever; a natural anti-inflammatory; and a natural anti-microbial agent. The topical sting/bite treatment compound can neutralize venom, debride the sting/bite wound site, relieve pain, reduce swelling and prevent infection.
Type:
Application
Filed:
June 11, 2015
Publication date:
December 15, 2016
Applicant:
BIOSTAT RESEARCH, LLC
Inventors:
William Fletcher Huggins, Joanne S. Walter
Abstract: A method for treating acute attacks of hereditary angioedema (HAE) whereby a first does and a second dose of a recombinant C1 esterase inhibitor is administered intravenously to the patient, each dose at 50 IU/kg body weight of the patient and wherein the first and second doses are administered within a 24 hour period. The recombinant C1 esterase inhibitor has an amino acid sequence identical to the amino acid sequence of human plasma-derived C1 esterase inhibitor and a modified carbohydrate structure as compared to the human plasma-derived C1 esterase inhibitor. Relief of attack symptoms as well as reduction of relapse and/or new attack symptoms are achieved by use of the method.
Abstract: Methods are described for preventing or reducing ischemia and/or systemic inflammatory response in a patient such as perioperative blood loss and/or systemic inflammatory response in a patient subjected to cardiothoracic surgery, e.g. coronary artery bypass grafting and other surgical procedures, especially when such procedures involve extra-corporeal circulation, such as cardiopulmonary bypass.
Type:
Application
Filed:
June 7, 2016
Publication date:
December 15, 2016
Applicant:
Dyax Corp.
Inventors:
Robert Charles Ladner, Arthur Charles Ley, Shirish Hirani, Anthony Williams
Abstract: A novel method of using an aquaporin protein from Rhipicephalus microplus (RmAQP2), fragments of RmAQP2, and/or the cDNA encoding RmAQP2 and/or the fragments are described. Immunogenic composition containing recombinant RmAQP2 and/or fragments of RmAQP2 are produced and administered to an ungulate which generates an immune response to RmAQP2. After feeding female ticks on the ungulate injected with RmAQP2 and/or fragments of RmAQP2, the female ticks have lower reproductive viability because of a reduced egg mass, reduced hatching percentage, and reduced survival of larvae. Thus, administering RmAQP2 and/or RmAQP2 fragments to an ungulate can reduce the incidence of R. microplus and also reduce the incidence of tick-borne pathogens in ungulates because of the lower number of R. microplus.
Type:
Application
Filed:
June 9, 2016
Publication date:
December 15, 2016
Inventors:
Glen A. Scoles, Felicito Guerrero, Reginaldo Bastos
Abstract: A composition for treating type 1 diabetes mellitus autoimmunity can include a therapeutically effective amount of two or more overlapping fragments of preproinsulin and a pharmaceutically acceptable carrier, wherein at least one of the polypeptide fragments is antigenic.
Abstract: The disclosure is in the field of immunology and provides means and methods for the treatment of multiple sclerosis. More in particular, the disclosure relates to means and methods for inducing immune tolerance to an antigen, wherein the antigen is covalently attached to a sialylated oligosaccharide, in particular, 6?-sialyl-N-acetyllactosamine. More in particular, the disclosure provides means and methods for inducing immune tolerance against an antigen derived from myelin, such as a myelin component, such as myelin oligodendrocyte glycoprotein, a well-known target in experimental autoimmune encephalomyelitis, a murine multiple sclerosis model.
Type:
Application
Filed:
June 10, 2015
Publication date:
December 15, 2016
Inventors:
Yvette Van Kooyk, Wendy Wilhelmina Josephina Unger
Abstract: Disclosed are methods, compositions of matter, and protocols useful for the induction of a therapeutic immune modulatory response through administration of exosomes derived from a stem cell source. In one embodiment, said stem cell source is endometrial regenerative cells. Specifically, in one embodiment stem cell derived exosomes are used as a method of treating an autoimmune condition such as rheumatoid arthritis, multiple sclerosis, or systemic lupus erythromatosis.
Abstract: Customized whole cell cancer vaccines can be produced from autologous (ex vivo or in situ) or allogeneic human or veterinary patient cell lines. Cells are transformed with S. pyogenes DNA that expresses an Emm protein on the cell surface and cytosol. Treatment of cancer patients with an Emm vector vaccine induces an immunologic response to the cancer by enhancing immunogenicity of a tumor. Emm vaccines can be used in patients where the cancer is not identified due to lower tumor burden or used to treat a specific cancer and subsequently treat for a second type that may have arisen through metastasis.
Type:
Application
Filed:
March 4, 2015
Publication date:
December 15, 2016
Inventors:
MICHAEL J. P. LAWMAN, PATRICIA D. LAWMAN, VIJAY RAMIYA
Abstract: This invention provides compositions and methods for treating and vaccinating against a HER2/neu antigen-expressing tumor and inducing an immune response against the same in a subject.
Type:
Application
Filed:
February 25, 2015
Publication date:
December 15, 2016
Inventors:
Vafa Shahabi, Anu Wallecha, Paulo C. Maciag, Yvonne Paterson, Nicola Mason, Matthew Seavey
Abstract: Materials and methods for identifying and using MEW molecule variants for activating self-reactive T cells in a peptide-specific manner, and their use to focus autoimmune cellular responses against diseases such as cancers and persisting viral infections, are described.
Abstract: Provided herein are consensus amino acid sequences of prostate antigens that are capable of breaking tolerance in a targeted species, including PSA, PSMA, STEAP and PSCA antigens. Also provided are nucleic acid sequences that encode one or more consensus amino acid sequences of prostate antigens PSA, PSMA, STEAP and PSCA, as well as genetic constructs/vectors and vaccines expressing the sequences. Also provided herein are methods for generating an autoimmune response against prostate cancer cells by administering one or more of the vaccines, proteins, and/or nucleic acid sequences that are provided.
Type:
Application
Filed:
July 11, 2016
Publication date:
December 15, 2016
Inventors:
David Weiner, Jian Yan, Bernadette Ferraro, Niranjan Y. Sardesai, Mathura P. Ramanathan
Abstract: The present invention relates to methods of suppressing the immune tolerance of a disease or disease antigens in a patient. The method also promotes the activity of the effectore T lymphocytes. The invention includes administering a therapeutic composition that promotes a Th1 environment in the patient while decreasing the immunosuppressive activity of Treg cells that can lead to disease antigen tolerance and immunoavoidance of the disease antigens by the patient. The therapeutic composition includes allogeneic emTh-1 cells. The therapeutic composition can also include disease antigens such as the chaperone-rich cell lysate of the disease antigen.
Abstract: The present invention concerns methods and compositions for forming anti-cancer vaccine DNL complexes using dock-and-lock technology. In preferred embodiments, the anti-cancer vaccine DNL complex comprises an antibody moiety that binds to dendritic cells, such as an anti-CD74 antibody or antigen-binding fragment thereof, attached to an AD (anchoring domain) moiety and a xenoantigen, such as CD20, attached to a DDD (dimerization and docking domain) moiety, wherein two copies of the DDD moiety form a dimer that binds to the AD moiety, resulting in the formation of the DNL complex. The anti-cancer vaccine DNL complex is capable of inducing an immune response against xenoantigen expressing cancer cells, such as CD138negCD20+ MM stem cells, and inducing apoptosis of and inhibiting the growth of or eliminating the cancer cells.
Abstract: The present invention relates to an isolated polypeptide antigen of Acinetobacter baumannii, comprising at least an amino acid sequence selected from the group consisting of: (a) SEQ ID NOs: 1-5; (b) an amino acid sequence having at least 80% sequence identity to SEQ ID NOs: 1, 3, 4 or 5; (c) an amino acid sequence having at least 60% sequence identity to SEQ ID NO: 2; and (d) a fragment of the amino acid sequence according to (a) to (c); wherein the polypeptide having the amino acid sequence according to (b) to (d) has immunostimulatory activity. The universal polypeptide antigens of Acinetobacter baumannii can be used in the universal vaccine preparation. The corresponding antibodies can be used in the diagnostic and treatment of Acinetobacter baumannii.
Abstract: The present disclosure relates generally to therapeutic compositions comprising recombinant bacteria. Further, the disclosure elaborates upon methods of utilizing the taught therapeutic compositions to treat autoimmune and inflammatory disease. The present teachings also relate to the disclosed recombinant bacteria and methods of producing the recombinant bacteria utilized in the compositions and methods. Further taught herein are dietary supplements and food additive compositions comprising the taught recombinant bacteria.
Abstract: Provided herein are chimeric influenza hemagglutinin (HA) polypeptides, compositions comprising the same, vaccines comprising the same, and methods of their use.
Type:
Application
Filed:
May 19, 2016
Publication date:
December 15, 2016
Applicant:
ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
Inventors:
Adolfo Garcia-Sastre, Peter Palese, Florian Krammer
Abstract: The present invention is based on the surprising finding that H5 protein of clade 1 H5N1 induces, in particular by a single-shot vaccination, a cross-clade protective immune response to influenza viruses with H5N1 HA. In one aspect, the invention is thus directed to H5 protein of clade 1 H5N1 virus for use in a method of treating or preventing infections with H5N1 virus of a different clade, wherein the H5N1 virus of a different clade comprises a particular polynucleotide and/or H5 protein.
Abstract: The present invention relates to compositions and methods for the prevention, treatment, and diagnosis of Hepatitis B virus (HBV) infection, and discloses peptides, polypeptides, and polynucleotides that can be used to stimulate a CTL response against HBV infection. The peptide and/or proteins of the invention may be used as a therapeutic drug to stimulate the immune system to recognize and eliminate HBV infection in infected cells or as a vaccine for the prevention of disease.
Abstract: The instant invention provides for novel lipid nanoparticle (LNP) formulations, containing cationic lipids, for use as vaccine adjuvants and/or as antigen delivery systems. It is an object of the instant invention to provide LNP formulations that demonstrate enhancements in humoral and cellular immunogenicity of vaccine antigens, particularly subunit vaccine antigens, when utilized alone or in combination with immunostimulatory agents (e.g. small molecule or oligonucleotide TLR agonists). The instant invention further identifies physical and chemical properties of the LNP formulations that can be manipulated to enhance antigen efficiency and adjuvant tolerability in vivo.
Type:
Application
Filed:
February 23, 2015
Publication date:
December 15, 2016
Applicant:
Merck Sharp & dohme Corp
Inventors:
Marian Gindy, Danilo R. Casimiro, Andrew Bett, Jan H. Ter Meulen
Abstract: The invention comprises a composition with means to inhibit the function of the inflammatory cytokine IL-17 and methods for using this composition to treat IL-17-mediated ocular inflammatory disorders. The invention also discloses devices for delivering this composition to the eye.
Abstract: The invention relates to a method of modulating concentration of Alzheimer's Disease (AD) relevant proteins amyloid precursor protein (APP), beta (?) and gamma (?) secretases and amyloid beta peptide (A?), and also relates to a method of reducing A? shedding. Furthermore, this invention extends to a compound for use in the treatment of AD, and also to a method of treating AD.
Type:
Application
Filed:
May 10, 2016
Publication date:
December 15, 2016
Applicant:
UNIVERSITY OF THE WITWATERSRAND, JOHANNESBURG
Inventors:
Stefan Franz Thomas Weiss, Katarina Jovanovic, Danielle Gonsavles, Bianca Da Costa Dias, Stefan Knackmuss, Uwe Reusch, Melvyn Little, Marc Saul Weinberg
Abstract: Methods of modulating immune responses in subjects having oncology- and immune-related diseases, disorders and conditions by the administration of an IL-10 agent, including pegylated IL-10.
Abstract: A composition includes an amount of potable water existing in a liquid crystalline state. The amount of potable water is resonant with a material. A measure of preservative is mixed with the amount of potable water existing in the liquid crystalline state.
Abstract: An example implantable microparticle for delivering therapeutic heat treatment comprises a generally spherical body. The body may be formed from a first material comprising a biodegradable material and a second material comprising a Curie temperature material. The biodegradable material may be a non-Curie temperature material or have a Curie temperature lower than a Curie temperature of the Curie temperature material. The first material and the second material are mixed to form a composite having a Curie temperature in the range of 35° C. and 100° C.
Type:
Application
Filed:
June 15, 2016
Publication date:
December 15, 2016
Inventors:
Derek C. Sutermeister, Martin R. Willard, Patrick A. Haverkost, Timothy A. Ostroot
Abstract: [Problem to be Solved] It is an object of the present invention to provide a preventive or therapeutic composition for a therapy-resistant cancer having therapy-resistant cancer cells. [Solution] A preventive or therapeutic composition for a therapy-resistant cancer having therapy-resistant cancer cells, which is for use in photodynamic therapy, wherein the composition comprises ALAs.
Type:
Application
Filed:
February 16, 2015
Publication date:
December 15, 2016
Applicants:
SBI Pharmaceuticals Co., Ltd., Osaka Medical and Pharmaceutical College
Abstract: Peroxides and chlorine dioxide (compounds) can be stabilized for long periods of time (years) by combining the compounds with water that has been infused with SG gas. Such stabilized materials can be used to infuse soft, solid substrates that can be used as sterile wipes, wound dressings, or the like.
Abstract: The present invention is related to methods for lowering peroxide levels in sucrose acetate isobutyrate formulations and to composition used in and formed by such methods.
Type:
Application
Filed:
August 29, 2016
Publication date:
December 15, 2016
Applicant:
DURECT CORPORATION
Inventors:
Gunjan Junnarkar, Michael A. Desjardin, John P. Carr
Abstract: The present invention provides implants comprising a therapeutic drug and a polymer containing polylactic acid (PLA) and optionally polyglycolic acid (PGA). The present invention also provides methods of maintaining a therapeutic level of a drug in a subject, releasing a therapeutic drug at a substantially linear rate, and treating schizophrenia and other diseases and disorders, utilizing implants of the present invention.
Abstract: The present invention relates to a pharmaceutical composition in the form of a storage-stable solution for the parenteral administration of ultrashort-effective ?-adrenoreceptor antagonists, comprising a) an ultrashort-effective ?-adrenoreceptor antagonist and/or a pharmaceutically acceptable salt thereof, b) water, and c) a cyclodextrin and/or a functional cyclodextrin derivative. The composition according to the invention has high stability, even without the presence of additional adjuvants.
Abstract: The invention relates to selected carbohydrate-lipid constructs and their use as mimics of ligands for receptors expressed by a virus. In particular, the invention relates to the use of selected carbohydrate-lipid constructs in methods of inhibiting virus infection and/or promoting clearance of virus from infected subjects. Carbohydrate-lipid constructs selected for use in these methods where the virus is Human Immunodeficiency Virus (HIV) are provided.
Abstract: Methods for modifying therapeutic agents such as therapeutic biomolecules, such as proteins for improved oral, rectal or transmucosal delivery, as well as compositions made using such methods and methods of administering such compositions to a subject, are disclosed. Specifically, the therapeutic agents are conjugated to hyperbranched polymers (HBPs), such as hyperbranched polyglycerol (HPG). When such conjugates are administered orally to a subject, the HBP protects the therapeutic agent from the acid environment of the stomach and protease attack in the gastro-intestinal tract, while facilitating the absorption of the therapeutic agent in the higher pH environment of the intestines. The methods and compositions are useful for the improved administration of a variety of therapeutic agents to a subject.
Type:
Application
Filed:
February 23, 2015
Publication date:
December 15, 2016
Inventors:
YUNFENG LU, Juanjuan Du, Jie Li, Yang Liu
Abstract: This invention relates to novel PEGx-GCSF conjugates, wherein x is the amount of PEG per GCSF and ranges from 4 to 8. The invention also relates to PEG[x]-GCSF populations of individual PEGx-GCSF conjugates, wherein [x] is the average amount of PEG per GCSF of the population and is 4 or greater. The inventive compositions have unexpected therapeutic efficacy, while avoiding or substantially reducing the likelihood of adverse side effects.
Type:
Application
Filed:
June 13, 2016
Publication date:
December 15, 2016
Inventors:
Abraham Abuchowski, Ronald G. Jubin, Peter J. Buontempo, Friedericke Kazo
Abstract: Conjugates between Factor IX and PEG moieties. are disclosed in the present application. The conjugates are linked via a glycosyl linking group interposed between and covalently attached to the peptide and the modifying group. Conjugates are formed from glycosylated peptides by the action of a glycosyltransferase. The glycosyltransferase ligates a modified sugar moiety onto a glycosyl residue on the peptide. Also provided are methods for preparing the conjugates, methods for treating various disease conditions with the conjugates, and pharmaceutical formulations including the conjugates.
Type:
Application
Filed:
August 19, 2016
Publication date:
December 15, 2016
Inventors:
Shawn Defrees, Robert J. Bayer, Caryn Bowe, Krishnasamy Panneerselvam
Abstract: The present invention provides methods to treating inflammation related disease and disorders such as an autoimmune disease and autoimmune related uveitis by administering compositions and formulations comprising MetAP-2 inhibitors as disclosed herein. The composition comprises a formulation of a fumagillol derivative that retains anti-inflammation activity and is associated with a block copolymer comprising a hydrophilic polymer moiety and a hydrophobic polymer moiety.
Type:
Application
Filed:
August 26, 2016
Publication date:
December 15, 2016
Applicant:
CHILDREN'S MEDICAL CENTER CORPORATION
Inventors:
Ofra Benny-Ratsaby, Robert D'Amato, Takeru Yoshimura
Abstract: A chimeric toxin is disclosed comprising a peptide ligand specifically targeting neurons involved in pain processing; and a clostridial neurotoxin light chain, wherein the ligand is linked to the light chain. The methods of preparing such chimeric toxin and the method of using the chimeric toxin to regulate pain transmission are also disclosed.
Type:
Application
Filed:
August 19, 2016
Publication date:
December 15, 2016
Inventors:
Eric A. Johnson, Sabine Pellett, William H. Tepp, Tony L. Yaksh, Marc Marino, Qinghao Xu
Abstract: The invention relates to materials and methods of conjugating a water soluble polymer to an oxidized carbohydrate moiety of a blood coagulation protein comprising contacting the oxidized carbohydrate moiety with an activated water soluble polymer under conditions that allow conjugation. More specifically, the present invention relates to the aforementioned materials and methods wherein the water soluble polymer contains an active aminooxy group and wherein an oxime linkage is formed between the oxidized carbohydrate moiety and the active aminooxy group on the water soluble polymer. In one embodiment of the invention the conjugation is carried out in the presence of the nucleophilic catalyst aniline. In addition the generated oxime linkage can be stabilized by reduction with NaCNBH3 to form an alkoxyamine linkage.
Type:
Application
Filed:
August 29, 2016
Publication date:
December 15, 2016
Inventors:
Juergen Siekmann, Stefan Haider, Hanspeter Rottensteiner, Peter Turecek
Abstract: Peptides that specifically bind erythrocytes are described. These are provided as peptidic ligands having sequences that specifically bind, or as antibodies or fragments thereof that provide specific binding, to erythrocytes. The peptides may be prepared as molecular fusions with therapeutic agents, tolerizing antigens, or targeting peptides. Immunotolerance may be created by use of the fusions and choice of an antigen on a substance for which tolerance is desired.
Type:
Application
Filed:
August 9, 2016
Publication date:
December 15, 2016
Inventors:
Jeffrey A. Hubbell, Stephan Kontos, Karen Y. Dane
Abstract: The invention described herein pertains to the diagnosis, imaging, and/or treatment of pathogenic cell populations. In particular, the invention described herein pertains to the diagnosis, imaging, and/or treatment of diseases caused by PSMA expressing cells, such as prostate cancer cells, using compounds capable of targeting PSMA expressing cells.
Type:
Application
Filed:
August 24, 2016
Publication date:
December 15, 2016
Inventors:
Iontcho Radoslavov VLAHOV, Joseph Anand REDDY, Alicia BLOOMFIELD, Ryan DORTON, Melissa NELSON, Marilynn VETZEL, Christopher Paul LEAMON
Abstract: The invention described herein pertains to the diagnosis, imaging, and/or treatment of pathogenic cell populations. In particular, the invention described herein pertains to the diagnosis, imaging, and/or treatment of diseases caused by PSMA expressing cells, such as prostate cancer cells, using compounds capable of targeting PSMA expressing cells.
Type:
Application
Filed:
August 24, 2016
Publication date:
December 15, 2016
Inventors:
Iontcho Radoslavov VLAHOV, Joseph Anand REDDY, Alicia BLOOMFIELD, Ryan DORTON, Melissa NELSON, Marilynn VETZEL, Christopher Paul LEAMON
Abstract: The present invention relates to a substrate for transglutaminase comprising a peptide having from 3 to 15 amino acids and comprising the amino acid sequence X?4X?3X?2X?1QX+1X+2X+3X+4, bound to a first molecule.
Abstract: The present invention concerns methods and compositions for forming PEGylated complexes of defined stoichiometry and structure. In preferred embodiments, the PEGylated complex is formed using dock-and-lock technology, by attaching a target agent to a DDD sequence and attaching a PEG moiety to an AD sequence and allowing the DDD sequence to bind to the AD sequence in a 2:1 stoichiometry, to form PEGylated complexes with two target agents and one PEG moiety. In alternative embodiments, the target agent may be attached to the AD sequence and the PEG to the DDD sequence to form PEGylated complexes with two PEG moieties and one target agent. In more preferred embodiments, the target agent may comprise any peptide or protein of physiologic or therapeutic activity. The PEGylated complexes exhibit a significantly slower rate of clearance when injected into a subject and are of use for treatment of a wide variety of diseases.
Type:
Application
Filed:
August 25, 2016
Publication date:
December 15, 2016
Inventors:
Chien-Hsing Chang, David M. Goldenberg, William J. McBride, Edmund A. Rossi
Abstract: The present invention relates to an improved method of providing photoreceptor function to a cell, for example for use in the treatment of retinal degeneration. The present invention also relates to compositions and kits, in particular for use in such methods.
Type:
Application
Filed:
February 24, 2015
Publication date:
December 15, 2016
Applicant:
The University of Manchester
Inventors:
Robert Lucas, Paul Bishop, Jasmina Cehajic-Kapetanovic
Abstract: The present invention relates to a pharmaceutical composition comprising a modified mRNA that is stabilised by sequence modifications and optimised for translation. The pharmaceutical composition according to the invention is particularly well suited for use as an inoculating agent, as well as a therapeutic agent for tissue regeneration. In addition, a process is described for determining sequence modifications that promote stabilisation and translational efficiency of modified mRNA of the invention.
Type:
Application
Filed:
August 26, 2016
Publication date:
December 15, 2016
Applicant:
CureVac AG
Inventors:
Florian VON DER MÜLBE, Ingmar HOERR, Steve PASCOLO
Abstract: Disclosed are capsid-mutated rAAV vectors and methods for their use in gene therapy, and particularly for use in delivering therapeutic transgenes to treat a variety of mammalian diseases and disorders, including dysfunctions and abnormal conditions of the human eye. VP3 capsid proteins comprising a modification of one or more of the surface-exposed tyrosine residues are disclosed, and in particular, VP3 capsid protein comprising tyrosine-to-phenylalanine mutations at positions corresponding to Y444F, Y500F, and Y730F of the wild-type AAV2 sequence. Also provided are rAAV virions and viral particles that comprise such a mutated AAV capsid protein and a nucleic acid molecule that expresses one or more selected therapeutic or reporter transgenes in one or more mammalian cells of interest. Advantageously, the capsid-mutated rAAV vectors and virions disclosed herein afford improved transduction efficiency in a variety of cells, tissues and organs of interest, when compared to their unmodified (i.e.
Type:
Application
Filed:
August 24, 2016
Publication date:
December 15, 2016
Inventors:
Mavis AGBANDJE-MCKENNA, William W. HAUSWIRTH, Arun SRIVASTAVA, Li ZHONG