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The Analyses and Estimates the CRISPR Market by: Type, End-Users, Geography SA-BRC is pleased to announce the initiation of research on "Global CRISPR Products and Services Market." Houston, TX, May 03, 2017 --( Rapid growth in biotechnology and advances in genetic engineering has accelerated life science research. Increasing understanding of genetic makeup of various life forms and their role has provided research across the globe with solid grounds to discover novel therapeutic and commercially viable alternatives. With current alarming rise in incidence of chronic diseases such as cancer, diabetes, and various other cardiovascular and neurological disorders, there is an urgent need of long term treatment substitute. Advances in genetic research such as the CRISPR technology would help in achieving these milestones earlier. Request Free Report Sample@ www.sa-brc.com/Global-CRISPR-Products-and-Services-Market-Assessment--Forecast-2017-2021/up100 Although low cost, and ease of use are beneficial for genetic research, scientists also believe that widespread and uncontrolled sale of such genetic engineering tools through e-commerce websites with increase the risk of bio-hacking. The CRISPR kits are available online at a price of less than US$ 200. This encourages uncontrolled and unauthorized use of biotechnology tools in modifying genetic sequences. Such research activities are likely to result in new disease causing agents that can affect large percentage of population globally. Various genetic researchers, advocacy groups and industry experts have demanded control of regulatory authorities for sale of CRISPR kits over e-commerce websites. Implementation of restraints on online sales would drastically affect the CRISPR market growth. Companies are seen investing heavily in providing cost efficient and CRISPR kits for more accurate results. In 2017, Synthego raised US$ 41 million to fortify its portfolio for CRISPR gene editing kits. Key players in the clustered regularly interspaced short palindromic repeats (CRISPR) market include ODIN, Thermo Fisher Scientific, Inc., OriGene Technologies, Inc., Cellecta, Inc., Takara Bio, System Biosciences, Inc., BioCat GmbH, Synthego Corporation, QIAGEN, Biolegio B.V. and various others. Request For TOC@ www.sa-brc.com/Global-CRISPR-Products--Services-Market-Assessment--Forecast-2017-2021/upcomingdetail100 Houston, TX, May 03, 2017 --( PR.com )-- Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) are partially palindromic repeated DNA sequences observed in bacteria. These segments act as an immune system for bacteria against virus infections. CRISPR has found application in gene editing due to high flexibility and specific targeting. The technology has been widely implemented in stem cell research, gene therapy research, tissue and animal disease models, and various others. These advances in genetic research have witnessed rapid market acceptance as large number of research institutes and government agencies invested heavily in advanced research including stem cell research, gene therapy research, and various other research activities aimed at development of novel therapeutic. Hence introduction of new technologies assisting in these research activities receives swift acceptance in the research community. Wide spread awareness about the technology and its application has driven the CRISPR market globally. Simplicity of the technology and ease of use are other factors driving the CRISPR market growth and the growth for CRISPR services. Genomic engineering in cell lines is a multipurpose tool for researching biopharmaceutical research, designing diseases models, gene function, drug discovery among several other applications.Rapid growth in biotechnology and advances in genetic engineering has accelerated life science research. Increasing understanding of genetic makeup of various life forms and their role has provided research across the globe with solid grounds to discover novel therapeutic and commercially viable alternatives. With current alarming rise in incidence of chronic diseases such as cancer, diabetes, and various other cardiovascular and neurological disorders, there is an urgent need of long term treatment substitute. Advances in genetic research such as the CRISPR technology would help in achieving these milestones earlier.Request Free Report Sample@ www.sa-brc.com/Global-CRISPR-Products-and-Services-Market-Assessment--Forecast-2017-2021/up100Although low cost, and ease of use are beneficial for genetic research, scientists also believe that widespread and uncontrolled sale of such genetic engineering tools through e-commerce websites with increase the risk of bio-hacking. The CRISPR kits are available online at a price of less than US$ 200. This encourages uncontrolled and unauthorized use of biotechnology tools in modifying genetic sequences. Such research activities are likely to result in new disease causing agents that can affect large percentage of population globally. Various genetic researchers, advocacy groups and industry experts have demanded control of regulatory authorities for sale of CRISPR kits over e-commerce websites. Implementation of restraints on online sales would drastically affect the CRISPR market growth.Companies are seen investing heavily in providing cost efficient and CRISPR kits for more accurate results. In 2017, Synthego raised US$ 41 million to fortify its portfolio for CRISPR gene editing kits. Key players in the clustered regularly interspaced short palindromic repeats (CRISPR) market include ODIN, Thermo Fisher Scientific, Inc., OriGene Technologies, Inc., Cellecta, Inc., Takara Bio, System Biosciences, Inc., BioCat GmbH, Synthego Corporation, QIAGEN, Biolegio B.V. and various others.Request For TOC@ www.sa-brc.com/Global-CRISPR-Products--Services-Market-Assessment--Forecast-2017-2021/upcomingdetail100 Click here to view the list of recent Press Releases from SA-BRC


Patent
Cellecta, Inc. | Date: 2014-01-13

Methods of obtaining a single cell expression profile from a target mammalian cell are provided. Aspects of the methods include contacting a cellular sample which includes the target mammalian cell with a packaged viral barcoded trans-splicing library including a plurality of barcoded trans-splicing constructs under transduction conditions, where a barcoded trans-splicing construct includes a trans-splicing element linked to a barcode element. The methods further include generating expression data from the resultant transduced target mammalian cell to obtain the single cell expression data from the target mammalian cell. Also provided are compositions, e.g., libraries and components thereof, which find use in practicing the methods.


Grant
Agency: Department of Health and Human Services | Branch: | Program: SBIR | Phase: Phase II | Award Amount: 999.60K | Year: 2012

The DNA Damage and Repair (DDR) signaling modules are among the most commonly deregulated genes in human tumors. Deficiencies in DDR pathways are believed to influence tumorigenic processes by promoting a mutator phenotype, which contributes to the acquisition of genetic lesions and fuels malignant transformation. As DDR networks are extensively rewired in cancer cells, the concept of synthetic sickness/lethality (SL) can be exploited to identify novel therapeutic target(s) for cancer. RNA interference(RNAi) currently makes it possible to use high-throughput functional genomic strategies for SL target identification. Unfortunately, while RNAi has opened new avenues for improving the drug discovery process, these avenues remain only potential opportunities until we develop robust RNAi screening technologies, which include experimental and bioinformatics tools for drug target discovery, validation and integration into operational cell-based models. To address these issues, as outlined in the 290 SBIRcontract proposal, the development of a novel orthogonal functional genomics platform based on validated lentiviral shRNA libraries to facilitate the discovery of SL molecular targets en masse will be required. Accordingly, the ultimate goal of the 290SBIR research project is to develop and commercialize a human pooled SL shRNA library that targets all of the canonical and non-canonical DDR (400x400) gene combinations and to validate their application in RNAi screens in cancer cell models. This project will also require the development of supporting tools, including a public SL DDR database, protocols, reagents and software tools for in vitro screening, and the validation of the SL hits that specifically control the proliferation and survival of cancer cells. The aforementioned genetic screening and bioinformatic tools will provide the research community with highly modular and cost-effective approaches to understand and integrate dynamic changes in DDR signaling networks for the discovery of novelanti-cancer SL targets.


Grant
Agency: Department of Health and Human Services | Branch: | Program: SBIR | Phase: Phase I | Award Amount: 299.95K | Year: 2013

Not Available


Grant
Agency: Department of Health and Human Services | Branch: | Program: SBIR | Phase: Phase II | Award Amount: 1.87M | Year: 2011

DESCRIPTION (provided by applicant): Targeted therapies have the ability to specifically interact with and block proliferation of cancer cells by interfering with targeted gene products required for the malignant phenotype. Unlike conventional cancer chemotherapies, targeted therapies do not harm normal cells of the body. This class of therapeutic has been met with considerable clinical success indicating that there is a great need for new targeted therapies that block progression of other types of cancerfor which no targeted therapy exists. We hypothesize that all hematopoietic cells (normal and cancerous) express genes responsible for maintaining the blood lineage and blood cancer phenotypes, and that the products of these genes can be exploited by newtargeted therapies. Identification of these hematopoieitc-specific lethal genes would allow for the development of novel targeted therapies for combating diverse blood cancers, and for developing tissue-specific, targeted conditioning regimens for hematopoietic stem cell transplantation with greatly reduced morbidity and mortality. We tested this hypothesis in the course of Phase I studies, and demonstrated that pooled shRNA genetic viability screens could be successfully used to identify novel lethality drug targets specific for hematopoietic cancer cell types. We also identified small molecule agents that exhibited hematopoietic-specificity closely correlating lethality profiles of the shRNAs identified in genetic screens. In order to translate this researches into the future development of novel classes of targeted therapies, we propose in the Phase II studies to concurrently apply genome-wide RNAi-based genetic screening and small molecule library high-throughput (HT) screens to identify lethality genesand cytotoxic small molecules specific for members of a diverse panel of hematopoetic cell lines. The hematopoieitc-specific lethal shRNAs, and small molecules targeting lethality genes will be further validated using a wide panel of hematopoietic and non-hematopoietic cell lines and normal PBMC. Similar toxicity/lethality profiles will allow us to prioritize cytotoxic compounds based on their mechanism of action and simultaneously identify their specific drug target(s). These studies will be extended to the lead compounds for targeted therapy of hematological cancers in Phase III of the project in a joint drug development project between Cellecta and Tartis, Inc. PUBLIC HEALTH RELEVANCE: The relevance of the proposed research to public health is that it may identify new drugs or biomolecules targetable by drugs, for treatment of blood cancers such as leukemia, or for new, reduced toxicity ablative drugs for stem cell transplantation conditioning.


Grant
Agency: Department of Health and Human Services | Branch: National Institutes of Health | Program: SBIR | Phase: Phase I | Award Amount: 224.91K | Year: 2015

DESCRIPTION provided by applicant Despite of rapid advances in elucidating the molecular basis of human diseases an ostensibly more difficult post genomic challenge is the functional annotation of disease specific signaling pathways and the integration of this information into the development of novel drugs Recent studies demonstrate that CRISPR CAS gene knock out technology can be used as a powerful tool for large scale functional genomic analysis in mammalian cells Unfortunately although the CRISPR CAS system has opened many potential avenues for improving the drug discovery process these avenues remain only potential opportunities until we develop robust commercially available CRISPR CAS screening technologies as well as experimental and bioinformatics tools for data validation and integration of this information into operational cell based models The ultimate goal of the proposed project is to develop a novel free public resource lentiviral sgRNA screening platform enabling scientists to perform genome wide CRISPR CAS genetic screens in a pooled format Specifically we propose using Phase I studies to prioritize the genetic screening performance of different sgRNA designs and to employ the best validated design to develop a K human sgRNA library targeting human genes Furthermore we will scale up the development and commercialization of a comprehensive set of human and mouse genome wide sgRNA libraries in Phase II studies These libraries will have improved performance and be designed for cost effective pooled format screening and identification of effectors by high throughput HT sequencing As supporting tools we will develop protocols reagents and software tools for screening data analysis and validation To test the performance of our functional genomics platform we propose to validate and compare our novel sgRNA and previously established shRNA resource to delineate the processes that underlie tumorigenesis in a panel of PDX derived lung cancer cell lines These newly developed sgRNA screening validation and software tools will be freely available for academic researchers through the open resource www decipherproject net site which was previously funded by NIH and developed by Cellecta for distributing genome wide shRNA libraries CRISPR CAS screening products and custom services will provide the research community with highly modular cost effective approaches for studies aimed at understanding and integrating dynamic changes in signal transduction networks and ultimately delineating disease specific phenotypes The proposed sgRNA screening and bioinformatics strategies harbor considerable potential to systematically identify new targets for therapeutic intervention and to facilitate the development of highly specific drugs biomarkers and novel therapeutic concepts PUBLIC HEALTH RELEVANCE Open Resource CRISPR CAS Genome Wide sgRNA Library Screening Platform The ultimate goal of the proposed project is to develop a novel free public resource lentiviral sgRNA screening platform enabling scientists to perform genome wide CRISPR CAS genetic screens in mammalian cells Through specific and irreversible inactivation one gene at the time in a pooled cell population of each protein encoding gene in the human genome researchers will be able to associate specific gene inactivation to specific phenotypes such as disease progression or regression greatly facilitating the discovery of new therapy targets We believe this new screening platform will provide a powerful tool for improving the drug discovery process Gene specificity in CRISPR CAS technology is achieved by small molecules called sgRNAs which can be designed to recognize any DNA sequence The two main goals of the proposed phase I project are to optimize the technology through a prioritization screen aimed at selecting the sgRNA design performing best in genetic screens to employ the best validated design to develop a sgRNA library targeting human genes In Phase we will scale up the development and commercialization of a comprehensive set of human and mouse sgRNA libraries to cover the whole protein encoding genome As supporting tools we will develop protocols reagents and software tools for screening data analysis and validation These developed sgRNA screening validation and software tools will be freely available for academic researchers through the open resource www decipherproject net site which was previously funded by NIH and developed by Cellecta for distributing genome wide shRNA libraries CRISPR CAS screening products and custom services will provide the research community with highly modular cost effective approaches for studies aimed at understanding and integrating dynamic changes in signal transduction networks and ultimately delineating disease specific phenotypes The proposed sgRNA screening and bioinformatics strategies harbor considerable potential to systematically identify new targets for therapeutic intervention and to facilitate the development of highly specific drugs biomarkers and novel therapeutic concepts


Grant
Agency: Department of Health and Human Services | Branch: | Program: SBIR | Phase: Phase II | Award Amount: 1.86M | Year: 2010

DESCRIPTION (provided by applicant): Despite rapid advances in elucidating the molecular basis of human diseases, an ostensibly more difficult post-genomic challenge is the functional annotation of disease-specific signaling pathways and integration of this information into the development of novel drugs. RNA interference (RNAi) now makes it possible to use large-scale functional genomic strategies for target identification. Unfortunately, while RNAi has opened many potential avenues for improving the drug discovery process, these avenues remain only potential opportunities until we develop robust RNAi screening technologies, as well as experimental and bioinformatics tools for data validation and integration of this information into operational cell-based models. To address these issues, in Phase I, we developed second generation functionally validated (FV) human druggable genome lentiviral 15K shRNA libraries, and we have demonstrated their utility for deciphering cell- signaling pathways. The ultimate goal of the Phase II studies is to develop and establish a cost-effective novel functional genomics platform to facilitate the discovery of therapeutic molecular targets en masse. Specifically, we propose to scale-up development of and commercialize a comprehensive set of human and mouse genome- wide FV shRNA libraries. These libraries will have improved performance and be designed for cost-effective pooled-format screening and identification of effectors by high-throughput (HT) sequencing. As supporting tools, we will develop protocols, reagents and software tools for in vitro and in vivo screening hit validation and therapeutic target prioritization. To test the performance of our functional genomics platform, we propose to use our novel RNAi resource to delineate the processes that underlie tumorigenesis in breast epithelial cells. We will perform synthetic lethality screens in a unique panel of isogenic human mammary epithelial cell (HMEC) lines that comprise the most relevant breast cancer genetic alterations. Furthermore, we will validate the results of our in vitro screens in xenograft models using both fully transformed HMECs and common breast cancer cell lines. Our findings will then be combined with data collected from scientific publications and presented in a publicly available knowledge base, with the ultimate goal of developing models of signaling pathways that specifically control the proliferation and survival of breast cancer cells. These developed RNAi screening, validation and software tools will be commercialized as products and custom services to provide the research community with highly modular, cost-effective approaches for studies aimed at understanding and integrating dynamic changes in signal transduction networks and ultimately delineating disease-specific phenotypes. As a result, we foresee that these toolsets will significantly improve the efficiency, economy and ease of elucidating and modeling disease-specific signal transduction networks and provide basic researchers with preferred, cost-effective alternatives to existing commercially available reagents and software. The proposed RNAi screening and bioinformatics strategies harbor considerable potential to systematically identify new anti-cancer targets for therapeutic intervention and to facilitate the development of highly specific drugs, biomarkers and novel therapeutic concepts. PUBLIC HEALTH RELEVANCE: The ultimate goal of the Phase II project is to develop and make commercially available a novel orthogonal functional genomics platform to facilitate discovery and validation of therapeutic molecular targets en masse. As a first step, we propose to develop and make commercially available a set of second generation of functionally-validated genome-wide human and mouse 65K pooled shRNA lentiviral libraries with improved performance and optimized design for cost-effective genetic screens. As a confirmation tool, we will develop protocols for high-throughput in-vitro and ex-vivo validation of drug target candidates identified in the screen with pooled shRNA sublibraries. From a bioinformatics viewpoint, we will make software tools for integration of RNAi screening data with transcriptome profiling and molecular network information mined from scientific literature. The proposed functional genomics platform will be applied and validated for the discovery of novel cancer therapeutic targets in a unique collection of isogenic human mammary epithelial cell (HMEC) lines, comprising the most common breast cancer genetic alterations. As a result of these studies we will reconstruct synthetic lethality pathways and make publicly available breast cancer knowledge database. These developed RNAi screening, validation and software tools will be commercialized as products and custom services to provide the research community with highly modular, cost-effective approaches for studies aimed at understanding and integrating dynamic changes in signal transduction networks and ultimately delineating disease-specific phenotypes. As a result, we foresee that these toolsets will significantly improve the efficiency, economy and ease of elucidating and modeling disease-specific signal transduction networks and provide basic researchers with preferred, cost-effective alternatives to existing commercially available reagents and software. The proposed RNAi screening and bioinformatics strategies harbor considerable potential to systematically identify new anti-cancer targets for therapeutic intervention and to facilitate the development of highly specific drugs, biomarkers and novel therapeutic concepts.


Grant
Agency: Department of Health and Human Services | Branch: | Program: SBIR | Phase: Phase I | Award Amount: 600.00K | Year: 2010

DESCRIPTION (provided by applicant): Systemic exposure to ionizing radiation (IR) results in cell damage in radiosensitive tissues. It is therefore highly desirable to develop efficient radioprotective agents for use in cancer radiotherapy, and biodefense. We have discovered a new class of radioprotective compounds derived from bacterial polypeptides that act as agonists of Toll-like receptors (TLRs). Our lead compound, CBLB502, binds to TLR5 and activates the NF-kB pro-survival pathway, which protects from IR damage. Phase I of this proposed research aims to improve CBLB502 and to identify novel radioprotective compounds from peptides that induce the NF-kB pathway by screening the libraries of BioActive Secreted Proteins (BASP). We will also derive high affinity CBLB502 BASP variants by mutagenesis. Potential radioprotective peptides that do not act through NF-kB will be isolated by direct functional selection for radiation protection. In addition, completion of Phase I of the proposed research will result in the development and commercialization of a new BASP screening technology, based on the in silico design and construction of short peptide BASP libraries with full control of amino acid residues, which is compatible with functional screening in disease-relevant cell models. This method was made possible by recent innovations in high-throughput (HT) chip-based oligonucleotide synthesis and HT sequencing technology. This simple and cost-effective approach represents an improvement on current drug discovery strategies and will accelerate the development of novel peptide therapeutics. Under Phase I funding, we propose to develop and validate this novel resource in vitro and in vivo. Given our unique interface with the cell-based drug screening facilities at the Roswell Park Cancer Center, we expect to develop a pipeline of potential drug peptide mimics with fast conversion of knowledge into efficient strategies of drug identification. Phase II of this research proposes an in-depth characterization of the isolated peptides as potential drugs. At the completion of the proposed studies, we anticipate to generate a novel class of radioprotective drugs. We believe the development of BASP screening platform will provide a valuable tool set for the identification and validation of novel biologically relevant peptide drugs. It will help to identify novel targets for therapeutic intervention and establish paradigms for drug discovery. Most importantly, this project will expedite the transition from bench to clinic and provide both academic and industrial researchers with a robust, cost-effective alternative to current peptide screening strategies. PUBLIC HEALTH RELEVANCE: The goal of this project is to identify novel radioprotective agents for biodefense and cancer treatment by peptide screening. The libraries of bioactive secreted peptides will be functionally screened for suppression of radiation-induced cell death and validated in vivo.


Sets of experimentally validated gene specific primer pairs are provided. Embodiments of the sets include 10 or more gene specific primer pairs of forward and reverse primers. The forward and reverse primers of each primer pair include gene specific primers that are experimentally validated as suitable for use in a multiplex amplification assay. In some instances, each of the forward and reverse primers includes an anchor domain that includes a universal primer binding site. The sets find use in a variety of different applications, including high-throughput sequencing applications.


Methods of clonal analysis of functional genomic assays are provided. Aspects of the invention include transducing a population of target cells with a packaged viral effector library made up of a plurality of effector construct subsets, wherein each effector construct subset of the library includes a plurality of effector constructs having a common effector cassette linked to a distinct clonal barcode. Inclusion of distinct clonal barcodes in the effector construct subset allows for determination of the clonal representation of an effector construct subset in transduced target cells that exhibit a specific phenotype. Aspects of the invention further include compositions, e.g., libraries and components thereof, which find use in practicing the methods.

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