However, scientists are still barely scratching the surface of the full capabilities of
gene targeting technology.
Not exact matches
The
technology's possibilities are staggering — in theory, allowing medical scientists to do everything from cure genetic disorders like sickle cell disease to identify
gene targets for combating HIV.
Well, one startup is seeking to take a very different approach: Exonics Therapeutics, which has secured $ 5 million in seed funding from CureDuchenne Ventures to see if the revolutionary new CRISPR - Cas - 9
gene - editing
technology can be used to
target the root genetic deficiency at the heart of the disease.
The Bill and Melinda Gates Foundation sees so much promise in
gene drive
technology that it plans to double spending on its
Target Malaria initiative, which aims to create systems for driving
genes in two species of malaria mosquitoes, to $ 70 million.
«We spent half a dozen years trying to
target the beta globin
gene using the old
technology,» he noted, adding that within one week of trying CRISPR, they had an editing tool that worked much better.
However, this
technology requires an exact match with any
targeted gene.
Researchers from KTH Royal Institute of
Technology's Science for Life Laboratory (SciLifeLab) research center and Gothenburg University employed the biological networks generated for 46 major human tissues in order to identify the liver - specific
gene targets.
The study adds to evidence that
gene editing may need to be adapted to each patient's genome, to ensure there aren't variants in DNA sequence in or near the
gene being
targeted that would throw off the
technology.
«So, with this knowledge, the dream is that we could eventually use
gene editing
technology to edit the microRNA
target sites in the host plants, preventing the microRNAs from binding and silencing these
genes.
He has worked in the biotech industry as a research scientist for over 11 years with a focus on emerging
technologies including
gene targeting in mice, molecular analysis of transgenes using GFP variants at the single cell level, and developing flow cytometry reagent kits to speed up assay development time for researchers.
Choudhury used high - throughput sequencing
technology to find the
targets of DAZAP1 and decipher whether DAZAP1 would bind to the exons and introns of
genes.
Derived mostly from human embryonic kidney 293T (HEK293T) and HeLa cell lines, EdiGene Knockout (KO) Cell Lysates have been optimized through the use of genome editing
technology and validated at the genomic level through PCR and Sanger - sequencing techniques to ensure the accuracy and knockout of the
target gene.
The convergence of several factors explains the trend: cheaper genetic sequencing
technologies, the discovery of new oncogenes (
genes that can cause a normal cell to become cancerous), a new generation of computers and bioinformatics that can analyze vast amounts of data, and a multibillion - dollar effort by researchers inside and outside the pharma industry to develop
targeted drugs and companion diagnostics for cancer.
The marriage of Professors Capecchi and Smithies» homologous recombination technique with Sir Martin's stem cell discoveries has created the highly versatile new
technology of
gene targeting.
Their research has created the
technology known as
gene targeting, now used in virtually all areas of biomedicine — from basic research to the development of new therapies.
However, like most
gene transfer approaches a major concern with ZFN
technology is the potential for oncogenesis due to off -
target effects.
First author Dr Reza Haqshenas said the researchers then used
gene silencing
technology to determine whether the
genes» cell factors identified using the antibody microarray were indeed important for HCV replication and therefore potential
targets for anti-HCV compounds.
Caribou's market - leading CRISPR - Cas9
gene editing
technology can accurately
target and cut DNA to produce precise and controllable changes to the genome, which can be applied by JAX to create mouse models that better recapitulate human diseases enabling researchers to find better treatments faster.
The advancement of the Cas9 - based platform for screening and validation will help further the development of new therapeutic products, and Caribou's CRISPR - Cas9
technology can utilize guide RNAs specific for unique sequences and
target a
gene at numerous sites and therefore provide enhanced specificity.
The CRISPR - Cas
technology can be used to screen for druggable
targets and to develop
gene and cell therapies for unmet medical needs.
Drug discovery for genetic diseases: Screening
technologies are applied to identify chemical compounds able to inhibit REST function, a major
target gene in Huntington's disease, and will serve as a paradigm for high - throughput screening approaches applied to neurological disorders.
This
technology helps researchers connect mutations in the so - called genomic «dark matter» with the
genes they affect, potentially revealing new therapeutic
targets for genetic disorders.
Intellia will utilize Caribou's proprietary CRISPR - Cas9
gene editing and repair
technology platform in the development of new therapies
targeting a variety of genetic - based diseases.
Karl Campbell from Island Conservation reported on his organization's investigation into using
gene - drive
technology as potentially the most
targeted, effective, and economic technique for eliminating invasive mice and rats.
use CRISPR - Cas
technology to carry out genome - wide screens of
gene -
gene,
gene - drug and cancer - microenvironment interactions in cells and mice in order to explore fundamental biology and to identify drug
targets and drug resistance / sensitisation mechanisms.
The CRISPR / Cas
technology applied to mouse genetic engineering could quickly advance scientific understanding of disease mechanisms by allowing researchers to ask complex questions and find answers much faster than with traditional
gene targeting approaches.
Developed in collaboration with the Laboratory Medicine, Information
Technology and Health Science Research departments of Mayo Clinic Geneticist Assistant NGS Interpretative Workbench, is a web - based tool for the control, visualization, interpretation and historical knowledge base of next generation sequencing data
targeted at specific
genes for the purpose of identifying potentially pathogenic variants associated with specific conditions such as hereditary colon cancer.
Scientific meeting «
Gene targeting, genome editing & transgenesis: research application» We are organizing joint meeting with our colleagues from Masaryk University in Brno dedicated to current progress in transgenic
technologies in research application, animal models of diseases and Wnt signalling.
«Simultaneous mutation detection, copy number measurement, and digital
gene expression profiling of high - grade serous ovarian cancer FFPE samples using Hyb & SeqTM
targeted sequencing
technology»
Same basic
technology, but
targeting a different
gene.
These knockout cell lines allow researchers to quickly validate their
gene or
target of interest, without having to invest time and resource in developing in - house CRISPR
technology.
This
technology can be applied to
gene expression, SNPs (single nucleotide polymorphisms) and proteomics data to improve diagnostic and drug
target identification and prioritization, predictive toxicology and drug screening.
This includes
targeting gene - editing
technologies like CRISPR, which has recently opened up new doors for precise genome manipulation and
gene therapy.
«With this
technology, you can test the function of these candidate
genes and measure what percentage of cells are generating into our
target arterial cells,» says Zhang.
He completed his postdoctoral studies in
gene targeting and DNA recombination at Massachusetts Institute of
Technology (MIT).
Participants will learn design of CRISPR
targets using bioinformatics tools, generation of
gene knockouts / knock - ins, and
target validation using the most current
technologies.
Title: Cross-Comparison of
Targeted Gene Expression
Technologies for Patient Stratification Date / Time: Tuesday, April 17 2018, 8:00 am - 12:00 pm CT Author: R. Venkatramanan et al, Covance Poster # / Location: 3418 / Section 16, Board 3 Hyperlink: http://www.abstractsonline.com/pp8/#!/4562/presentation/7089 A comparison of PlexSet
technology with various qPCR
technologies on 96 colorectal FFPE samples.
To repair the problem, the research team «broke» the mutated version of the MYPBC3
gene inside human embryos, using
technology that allows scientists to snip a specific
target sequence on a mutant
gene.