Abstract: The disclosure relates to omega-hydroxylase-related fusion polypeptides that result in improved omega-hydroxylated fatty acid derivative production when expressed in recombinant host cells. The disclosure further relates to microorganisms for ex pressing the omega-hydroxylase-related fusion polypeptides for the production of omega-hydroxylated fatty acid derivatives.
Type:
Application
Filed:
June 16, 2015
Publication date:
May 25, 2017
Applicant:
REG Life Sciences, LLC
Inventors:
Baolong ZHU, Andreas W. Schirmer, Cindy CHANG
Abstract: The invention relates to mTORbeta, a splice form of mTOR, nucleic acids encoding mTOR beta, and antibodies against mTOR beta. The invention also relates to methods of producing mTOR beta and methods of screening for an agent that modulates mTOR beta expression and/or activity. The invention further relates to a method of treating a disease associated with aberrant expression of mTOR beta by administration of an agent that alters mTOR activity and/or expression.
Type:
Application
Filed:
February 2, 2017
Publication date:
May 25, 2017
Applicants:
Ludwig Institute for Cancer Research, UCL Business PLC
Inventors:
Ivan Nemazanyy, Ganna Panasyuk, Alexander Zhyvoloup, Michael Waterfield, Ivan Gout
Abstract: The present invention relates to polymerase HBV mutant polypeptides comprising a mutated polymerase domain which is functionally disrupted for polymerase activity and fusion proteins comprising such polymerase mutant polypeptide. The present invention also relates to a nucleic acid molecule and an expression vector for expressing said polymerase mutant polypeptide as well as a composition which can be used for eliciting an immune response to HBV with the goal of providing a protective or therapeutic effect against HBV infection.
Abstract: The present invention provides a novel fusion polypeptide containing a catalytic domain of NPP1 fused to a targeting moiety, nucleic acids encoding the fusion polypeptide, a vector containing the nucleic acid integrated thereinto, a host cell transformed with the vector and pharmaceutical compositions comprising the fusion polypeptide.
Type:
Application
Filed:
January 27, 2017
Publication date:
May 25, 2017
Inventors:
Anthony QUINN, Alex J. HARVEY, Zhinan XIA
Abstract: The present invention relates to isolated polypeptides having xylanase activity and isolated polynucleotides encoding the polypeptides. The invention also relates to nucleic acid constructs, vectors, and host cells comprising the polynucleotides as well as methods of producing and using the polypeptides.
Type:
Application
Filed:
December 13, 2016
Publication date:
May 25, 2017
Inventors:
Elena Vlasenko, Brett McBrayer, Dominique Skovlund, Sara Landvik
Abstract: The present disclosure is generally directed to enzymes and in particular beta-glucosidase variants. Also described are nucleic acids encoding beta-glucosidase variants, compositions comprising beta-glucosidase variants, methods of using beta-glucosidase variants, and methods of identifying additional useful beta-glucosidase variants.
Type:
Application
Filed:
August 29, 2016
Publication date:
May 25, 2017
Applicant:
DANISCO US INC.
Inventors:
Richard R. BOTT, Thijs KAPER, Bradley R. KELEMEN, Frits GOEDEGEBUUR, Ronaldus Wilhelmus Joannes HOMMES, Slavko KRALJ, Paulien KRUITHOF, Igor NIKOLAEV, Wilhelmus Antonious Hendricus VAN DER KLEY, Johannes Franciscus Thomas VAN LIESHOUT, Sander VAN STIGT THANS, Gudrun VOGTENTANZ, Mats SANDGREN
Abstract: The present invention relates to isolated polypeptides having xylanase activity, catalytic domains, carbohydrate binding modules and polynucleotides encoding the polypeptides, catalytic domains or carbohydrate binding modules. The invention also relates to nucleic acid constructs, vectors, and host cells comprising the polynucleotides as well as methods of producing and using the polypeptides, catalytic domains or carbohydrate binding modules.
Abstract: Compositions comprising deimmunized lysostaphin and methods of using the same, e.g., to treat microbial infection in or on a subject, are provided.
Type:
Application
Filed:
May 14, 2015
Publication date:
May 25, 2017
Applicant:
TRUSTEES OF DARTMOUTH COLLEGE
Inventors:
Karl E. GRISWOLD, Chris BAILEY-KELLOGG, Yoonjoo CHOI, Kristina BLAZANOVIC, Hongliang ZHAO
Abstract: The present invention relates to a method for purifying a non-spreading botulinum toxin that causes local muscle paralysis and a non-spreading botulinum toxin obtained thereby. The method comprises the steps of: subjecting a purified botulinum toxin type A product to ion-exchange chromatography using a controlled pH of buffer, concentration of sodium chloride (NaCl), thereby separating the botulinum toxin type A product into subtractions; and collecting a subfraction having an A260/A280 value in a specific range from the separated subfractions.
Abstract: The present invention relates to novel subtilase variants exhibiting increased stability and preferably on par or improved wash performance. The variants of the invention are suitable for use in e.g. cleaning or detergent compositions, such as laundry detergent compositions and dish wash compositions, including automatic dish wash compositions. The present invention also relates to isolated DNA sequences encoding the variants, expression vectors, host cells, and methods for producing and using the variants of the invention.
Type:
Application
Filed:
July 6, 2015
Publication date:
May 25, 2017
Applicant:
NOVOZYMES A/S
Inventors:
Frank Winther Rasmussen, Peter Kamp Hansen, Lars Lehman Hylling Christensen
Abstract: The present invention provides a mutant with enhanced secretion of L-asparaginase and its application, which relates to the field of enzyme engineering. This invention successfully constructed a mutant with enhanced secretion of L-asparaginase through N-terminal deletion on amino acid sequence of L-asparaginase, and then expressed the mutant via a recombinant strain. Compared with the wild L-asparaginase, the secretion ability of L-asparaginase mutant in present invention significantly improves by 3.14 times compared to that of wild type. The recombinant strain that constructed in this prevention has an L-asparaginase yield of 407.6 U/mL and a production efficiency of 9.26 U/(mL/h), which reaches to the highest yield been reported.
Type:
Application
Filed:
June 22, 2016
Publication date:
May 25, 2017
Inventors:
Song LIU, Yue FENG, Jian CHEN, Guocheng DU, Yun JIAO, Jie RUAN, Hongye Cheng, Hui GAO
Abstract: The present invention relates to pharmaceuticals and modified beta-lactamases. Specifically, the invention relates to novel recombinant beta-lactamases and pharmaceutical compositions comprising the beta-lactamases. Also, the present invention relates to methods for modifying a beta-lactamase, producing the beta-lactamase and treating or preventing beta-lactam antibiotic induced adverse effects. Furthermore, the present invention relates to the beta-lactamase for use as a medicament and to the use of the beta-lactamase in the manufacture of a medicament for treating or preventing beta-lactam antibiotics induced adverse effects. Still further, the invention relates to a polynucleotide and a host cell comprising the polynucleotide.
Type:
Application
Filed:
January 27, 2017
Publication date:
May 25, 2017
Inventors:
Pertti Koski, Ulla Airaksinen, Katja Valimaki
Abstract: The expression plasmid vectors comprise a polynucleotide sequence encoding Ldc2 polypeptide, a fragment, and/or a mutant. A backbone plasmid is capable of autonomous replication in a host cell. The host cell is not a P. aeruginosa cell. Transformants are transformed with expression plasmid vector. The transformants are not P. aeruginosa. Mutant host cells comprise a polynucleotide sequence encoding Ldc2 polypeptide, a fragment and/or a mutant that has been integrated into the host cell chromosome. A polypeptide, a fragment and/or a mutant comprise Ldc2. A non-naturally occurring polynucleotide, and/or a mutant encodes polypeptide comprising Ldc2. Biobased cadaverine is produced using the transformants and the biobased cadaverine is prepared by the method. Polyamides are formed using the biobased cadaverine and compositions.
Type:
Application
Filed:
June 26, 2014
Publication date:
May 25, 2017
Applicants:
CATHAY R&D CENTER CO., LTD., CATHAY INDUSTRIAL BIOTECH LTD.
Abstract: Biomass (e.g., plant biomass, animal biomass, and municipal waste biomass) is processed for use in the production of useful products, such as fuels. For example, systems can use biomass materials, such as cellulosic and/or lignocellulosic materials, to enhance the production of a product, e.g., the production of ethanol and/or butanol by fermentation.
Type:
Application
Filed:
February 3, 2017
Publication date:
May 25, 2017
Inventors:
Marshall Medoff, Thomas Craig Masterman, Harrison Medoff
Abstract: The invention relates to a clustered regularly interspaced short palindromic repeats (CRISPR)/Cas guide RNA (gRNA) comprising a targeting domain that is complementary to human genomic Epidermal Growth Factor Receptor (EGFR) DNA, and a vector system including one or more packaged vector(s) including: (a) a first regulatory element operably linked to a gRNA, and (b) a second regulatory element operably linked to a nucleic acid encoding a Cas protein. Also disclosed are methods of altering a nucleic acid sequence encoding EGFR in a cell including contacting the cell with a vector system, methods of treating lung cancer, and methods of selectively inducing apoptosis in a cell including administering a gRNA to the cell.
Abstract: The invention generally relates to conducting an assay on a sample that isolates a bacterium from the sample, in which the assay isolates as low as about 1 CFU/ml of bacteria in the sample.
Type:
Application
Filed:
February 6, 2017
Publication date:
May 25, 2017
Inventors:
Victor C. Esch, Sergey A. Dryga, Lisa-Jo Ann Clarizia
Abstract: The present invention relates to methods for increasing the diversity of monoclonal antibodies produced against an antigen. The methods of the invention utilize immunization of a murine host defective in one or more enzymes involved in a post-translational modification of a polypeptide or a modification of a lipid, wherein said modification is exposed on a cell surface. The invention also relates to monoclonal antibodies produced by these methods and which are not produced when a normal mouse is immunized with the same antigen. The invention further relates to compositions comprising these monoclonal antibodies, as well as to such monoclonal antibodies bound or conjugated to a toxin, a detectable marker or to a solid support.
Abstract: The invention relates to a method for producing a chromosome substitution library, comprising: a) producing a population of DH lines from an at least partially heterozygous starting organism; b) identifying a first DH line in the population that has at least one non-recombined chromosome pair; c) identifying a matching second DH line in the population that has at least one non-recombined chromosome pair that is the same as the non-recombined chromosome pair of the first DH line, and at least one non-recombined chromosome pair that is different from the non-recombined chromosome pair of the first DH line; d) selecting the first DH line and the second DH line as a pair of parents, the progeny of which has at least three non-recombined chromosomes, at least two of which form a pair; e) repeating steps b), c) and d) one or more times on the DH population of the heterozygous starting organism, or repeating steps a)-d) on a DH population produced from a progeny plant of a pair of parents selected in step d), until
Abstract: The present invention provides novel bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom. Incorporation of one or more of the bicyclic carbocyclic nucleosides into an oligomeric compound is expected to enhance one or more properties of the oligomeric compound. In certain embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in modulation of normal function of the target RNA. In certain embodiments, bicyclic carbocyclic nucleosides are provided as monomers for use as antivirals.
Abstract: The present invention relates to a method of preparing triphosphate-modified oligonucleotides using a capture tag. The method allows the synthesis and purification of triphosphate-modified oligonucleotides in high yield and purity suitable for pharmaceutical applications.
Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Nuclear factor (erythroid-derived 2)-like 2 (NRF2), in particular, by targeting natural antisense polynucleotides of Nuclear factor (erythroid-derived 2)-like 2 (NRF2). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of NRF2.
Type:
Application
Filed:
September 20, 2016
Publication date:
May 25, 2017
Applicant:
CuRNA, Inc.
Inventors:
Joseph COLLARD, Olga KHORKOVA SHERMAN, Carlos COITO
Abstract: The present invention is directed to compounds, compositions, and methods useful for modulating NLRP3 mRNA or protein expression using gene silencing compounds comprising two or more single stranded antisense oligonucleotides that are linked through their 5?-ends to allow the presence of two or more accessible 3?-ends.
Type:
Application
Filed:
November 2, 2016
Publication date:
May 25, 2017
Inventors:
Weiwen Jiang, Mallikarjuna R. Putta, Fu-Gang Zhu, Jillian M. DiMuzio, Lakshmi Bhagat, Sudhir Agrawal
Abstract: In certain embodiments, methods, compounds, and compositions for treating B-cell lymphoma or hepatocellular carcinoma by inhibiting expression of ST AT3 mRNA or protein in an animal are provided herein. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate B-celllymphoma or hepatocellular carcinoma. The STAT (signal transducers and activators of transcription) family of proteins are DNA-binding proteins that play a dual role in signal transduction and activation of transcription.
Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Frataxin (FXN), in particular, by targeting natural antisense polynucleotides of Frataxin (FXN). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of FXN.
Abstract: The invention provides interfering RNA molecule-ligand conjugates useful as a delivery system for delivering interfering RNA molecules to a cell in vitro or in vivo. The conjugates comprise a ligand that can bind to a transferrin receptor (TfR). Therapeutic uses for the conjugates are also provided.
Abstract: Methods of treating complement-mediated ocular disorders by administering agents that inhibit a subject's complement component in an amount sufficient to treat the ocular disorder wherein, in a selected embodiment, said agent is an anti-complement aptamer that, in a preferred embodiment, is an anti-C5 aptamer.
Abstract: The invention provides novel immune regulatory oligonucleotides (IRO) as antagonist of TLRs and methods of use thereof. These IROs have unique sequences that inhibit or suppress TLR-mediated signaling in response to a TLR ligand or TLR agonist. The methods may have use in the prevention and treatment of cancer, an autoimmune disorder, airway inflammation, inflammatory disorders, infectious disease, skin disorders, allergy, asthma or a disease caused by a pathogen.
Abstract: The present invention provides a method for treating a mammalian subject affected by prostate cancer comprising i) an oligonucleotide which reduces clusterin expression and ii) a Heat Shock Protein 90 (Hsp90) inhibitor each in an amount that when in combination with the other is effective to treat the mammalian subject. The present invention also provides pharmaceutical compositions comprising an amount of an oligonucleotide which reduces clusterin expression, and a Hsp90 inhibitor for use in treating a mammalian subject affected by prostate cancer. Also provided are oligonucleotides which reduce clusterin expression for use in combination with a Hsp90 inhibitor in treating a mammalian subject affected by prostate cancer, and a composition for treating a mammalian subject affected by prostate cancer comprising i) an oligonucleotide which reduces clusterin expression and ii) a Hsp90 inhibitor each in an amount that when in combination with the other is effective to treat the mammalian subject.
Type:
Application
Filed:
May 23, 2016
Publication date:
May 25, 2017
Applicant:
The University of British Columbia
Inventors:
Martin E. GLEAVE, Amina ZOUBEIDI, Francois LAMOUREUX
Abstract: Some embodiments of the present invention relate to methods and compositions for treating cancer. More embodiments include methods and compositions for modulating the activity of the Hedgehog pathway.
Type:
Application
Filed:
November 30, 2016
Publication date:
May 25, 2017
Inventors:
Lalita Samant, Rajeev Samant, Shamik Das, Eddie Reed
Abstract: The invention provides compositions and methods for regulating microRNA (miRNA) biogenesis. The invention also relates to compositions and methods for treating or preventing cancer in a subject in need thereof.
Type:
Application
Filed:
January 31, 2017
Publication date:
May 25, 2017
Applicant:
PRESIDENT AND FELLOWS OF HARVARD COLLEGE
Abstract: The present invention relates to a method for predicting a survival rate of a breast cancer patient, comprising: providing a biological sample of the breast cancer patient; measuring miR-125a-5p expression level in the biological sample; and comparing the miR-125a-5p expression level in the biological sample of the breast cancer patient with miR-125a-5p expression level in another biological sample of a reference breast cancer patient; wherein when the miR-125a-5p expression level in the biological sample of the breast cancer patient is lower than that of the reference breast cancer patient, the survival rate of the breast cancer patient is lower than that of the reference breast cancer patient; or when the miR-125a-5p expression level in the biological sample of the breast cancer patient is greater than that of the reference breast cancer patient, the survival rate of the breast cancer patient is greater than that of the reference breast cancer patient.
Abstract: Disclosed are specific aphid dsRNA constructs that target either Chloride Intracellular Channel (CLIC) gene expression or Sucrase gene expression. Also disclosed is the use of dsRNA constructs of a CLIC gene to interfere with critical functions of CLIC gene peptide products. A novel method to develop nucleic acid control for pest management is also disclosed. Also disclosed is the use of dsRNA constructs to interfere with critical functions of Sucrase gene peptide products.
Abstract: The present invention relates to the use of modulators of Mcoln-1 for modulating the cells migration, in particular the migration of dendritic cells and tumor cells, especially for antitumoral vaccination, autoimmune diseases treatment, and metastasis prevention.
Type:
Application
Filed:
February 10, 2015
Publication date:
May 25, 2017
Inventors:
ANA-MARIA LENNON-DUMENIL, PABLO VARGAS, MARINE BRETOU
Abstract: Provided herein are compositions and methods for non-parenteral delivery of conjugated oligomeric compounds. In certain embodiments, compositions and methods are provided for oral delivery of conjugated oligomeric compounds. In certain embodiments, the oligomeric compounds are conjugated to one or more N-acetylgalactosamines or N-acetylgalactosamine analogues.
Type:
Application
Filed:
June 8, 2015
Publication date:
May 25, 2017
Applicant:
Ionis Pharmaceuticals, Inc.
Inventors:
Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Stanley T. Crooke
Abstract: The invention relates to bacterial artificial chromosomes (BAC) comprising retroviral nucleic acid sequences encoding: gag and pol proteins, and an env protein or a functional substitute thereof, wherein each of the retroviral nucleic acid sequences are arranged as individual expression constructs within the BAC. The invention also relates to uses and methods of transient transfection using said BAC.
Abstract: The invention discloses a promoter which can be induced to express in acidic conditions, and relates to the field of bioengineering technology. The promoters of the invention are separated from A. niger and can actuate and/or regulate the expression of the effectively connected nucleic acids in A. niger. In the invention the expression of the promoters is studied in A. niger, and it is indicated that some promoters show weak expression, and some show strong activity. The invention provides an effective method and new thought for organic acids production by fungi or other products produced by fermentation under acidic conditions.
Type:
Application
Filed:
November 11, 2016
Publication date:
May 25, 2017
Inventors:
Long Liu, Jian Chen, Guocheng Du, Xian Yin, Jianghua Li
Abstract: Provided herein compositions and methods for producing isoprenoids, including squalene. In certain aspects and embodiments provided are genetically converted yeast and uses therefore. In some aspects and embodiments, the genetically converted yeast produce isoprenoids, preferably squalene. Also are provided methods of producing squalene using a genetically converted yeast or a non-genetically converted yeast. The invention also provides squalene produced by genetically converted yeast or non-genetically converted yeast.
Type:
Application
Filed:
January 9, 2017
Publication date:
May 25, 2017
Inventors:
Keith A. Walker, Mark E. Knuth, Noel M. Fong, Peter R. Beetham
Abstract: Provided is a transgenic plant production method for producing a genetically modified rubber-producing plant that is capable of efficient short-term gene transfer, of introducing a target gene while retaining the useful traits of a plant undergoing gene transfer, and of efficient redifferentiation.
Abstract: In one aspect, there is provided a mutant, non-naturally occurring or transgenic plant cell comprising: (i) a polynucleotide comprising, consisting or consisting essentially of a sequence encoding a member of the CLC family of chloride channels and having at least 60% sequence identity to SEQ ID NO:1 or SEQ ID NO:2 or SEQ ID NO:3 or SEQ ID NO:4 or SEQ ID NO:10 or SEQ ID NO:11; (ii) a polypeptide encoded by the polynucleotide set forth in (i); (iii) a polypeptide comprising, consisting or consisting essentially of a sequence encoding a member of the CLC family of chloride channels and having at least 60% sequence identity to SEQ ID NO:5 or SEQ ID NO:6 or SEQ ID NO:7 or SEQ ID NO:12 or SEQ ID NO:13 or SEQ ID NO: 14; or (iv) a construct, vector or expression vector comprising the isolated polynucleotide set forth in (i); and wherein the expression or activity of the polynucleotide or the polypeptide is modulated as compared to a control plant and wherein the nitrate levels in the mutant, non-naturally occurring
Abstract: Two genes, A622 and NBB1, can be influenced to achieve a decrease of nicotinic alkaloid levels in plants. In particular, suppression of one or both of A622 and NBB1 may be used to decrease nicotine in tobacco plants.
Abstract: Compositions and methods include genetically encoding and expressing a novel delta-9 desaturase in plant cells. In some embodiments, methods of expressing nucleic acids in a plant cell to take advantage of the delta-9 desaturase enzyme's activity, such that the percent composition of saturated fatty acids in plant seeds is decreased and there is a concomitant increase in ?-7 fatty acids. In other embodiments, amino acid sequences have delta-9 desaturase activity. Methods can involve expression of delta-9 desaturase in plant cells, plant materials, and whole plants for the purpose of increasing the amount of unusual fatty acids in whole plants, plant seeds, and plant materials, for example, seeds.
Type:
Application
Filed:
December 15, 2016
Publication date:
May 25, 2017
Inventors:
Ann Owens MERLO, Daniel J. Gachotte, Mark A. Thompson, Terence A. Walsh
Abstract: Polynucleotide sequences encoding diacylglycerol acyltransferase genes and the use of these acyltransferases for increased seed storage lipid production and altered fatty acid profiles in oilseed plants are disclosed. Transgenic soybean seed having increased total fatty acid content of at least 20% and altered fatty acids when compared to the total fatty acid content of non-transgenic, null segregant soybean seed are described. Methods for increasing the total fatty acid content of a soybean seed by at least 20% include steps of transformation and selection.
Abstract: The invention relates to methods of producing a desired phenotype in a plant by manipulation of gene expression within the plant. The method relates to means which increase the level of expression of a MYB-subgroup14 polynucleotide or a MYB68 polypeptide. The method also relates to expression of a nucleic acid sequence encoding a MYB-subgroup14 or a MYB68 transcriptional factor. The methods are directed to elevating the levels of a MYB-subgroup14 or a MYB68 expression, wherein a desired phenotype such as reduced flower abortion and increased yield during heat stress is observed. The invention also relates to nucleic acid sequences useful in such methods.
Type:
Application
Filed:
January 27, 2017
Publication date:
May 25, 2017
Inventors:
Jiangxin Wan, Yafan Huang, Monika M. Kuzma
Abstract: The present invention provides for compositions and methods for producing sorghum crop plants that are resistant to herbicides. In particular, the present invention provides for sorghum plants, plant tissues and plant seeds that contain altered acetolactate synthase (ALS) genes and proteins that are resistant to inhibition by herbicides that normally inhibit the activity of the ALS protein.
Type:
Application
Filed:
January 26, 2017
Publication date:
May 25, 2017
Applicant:
Kansas State University Research Foundation
Abstract: The invention relates to a resistance gene and functional homologues or fragments thereof isolated from S. chacoense, S. berthaultii, S. sucrense or S. tarijense. Moreover, the invention relates to the use of said resistance gene, for example the use of said resistance gene in a method to increase or confer at least partial resistance in a plant to an oomycete infection. The invention provides an isolated or recombinant nucleic acid sequence comprising a nucleic acid sequence encoding one of the amino acid sequences of FIG. 4 or a functional fragment or a functional homologue thereof such as those presented in FIG. 13.
Type:
Application
Filed:
October 14, 2016
Publication date:
May 25, 2017
Applicant:
WAGENINGEN UNIVERSITEIT
Inventors:
Jacobus Hubertus VOSSEN, Maarten NIJENHUIS, Marion Johanna Barbara ARENS-DE REUVER, Edwin Andries Gerard VAN DER VOSSEN, Evert JACOBSEN, Richard Gerardus Franciscus VISSER
Abstract: Disclosed are delivery platforms for use in gene editing that include a relatively short, highly efficient promoter that drives transcription of a nucleic acid sequence that encodes a gene-editing molecule, e.g., either a gRNA or a nuclease. In conjunction with this promoter, the vector includes one or more transcription factor binding elements (an Sp1 binding element and/or an NF-?B binding element) cloned into the vector upstream of a promoter that drives transcription of a gene-editing molecule. The vector can be a all-in-one CRISPR/Cas9 delivery platform and can incorporate one or more of the transcription factor binding elements upstream of a promoter for the gRNA component and/or of a promoter for the nuclease component.
Abstract: Some aspects of this invention provide nucleic acid constructs for transgene expression. Some aspects of this invention provide multicistronic nucleic acid constructs, for example, comprising an expression cassette encoding a hairpin RNA and a reporter expression cassette. Some aspects of this invention provide nucleic acid constructs comprising two or more self-complementary nucleic acid sequences, for example, hairpin RNA encoding nucleic acid sequences and AAV inverse terminal repeats. Methods for the use of the constructs in therapy and research are also provided.