Sentences with phrase «gene targeting approaches»

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.

Not exact matches

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.

This study took a different approach — targeting a drug to a protein that controls how genes are turned on and off, early in the viral replication process.

«What we decided to do was take a completely unbiased approach where instead of targeting individual genes of interest, we would express randomized guides inside of the cell,» Lu says.

The findings by a team of Massachusetts General Hospital (MGH) investigators, which will be published in the April 24 issue of Cell and are receiving advance online release, support the importance of epigenetics — processes controlling whether or not genes are expressed — in cancer pathology and identify molecular circuits that may be targeted by new therapeutic approaches.

Most therapeutic approaches ignore the dynamic molecular network of genes, targeting instead only very few selected disease - related genes.

However, they determined the blood levels of SMN are not altered when SMN levels are altered in motor neurons using a gene therapy approach delivered directly into the cerebrospinal fluid to target the central nervous system.

Earlier MD Anderson investigations demonstrated exosomes as a factor in detecting pancreatic cancer, but these latest findings reveal genetically altered exosomes as a potentially novel approach for direct and specific targeting of mutated KRAS, the cancer gene commonly linked to pancreatic cancer.

The proposed strategy reflects a continuing move toward combination therapies, particularly when it comes to epigenetic approaches, which target the mechanisms that control whether genes are switched «on» or «off.»

«The trial is important for gene research and may be a proof of principle that targeting the most common mutation in cystic fibrosis is an effective approach for treatment of most patients,» Gentzsch said.

Despite this new direction for identifying targets for pharmaceutical interventions against RP, the researchers underscore that gene therapy still has great potential and possible benefits, and they are actively pursuing efforts with this approach for several forms of RP.

«Depending on the research questions, it may also be important to define the appropriate strategy to use, either whole - genome sequencing or just targeting some genomic regions with a gene baiting approach,» says Besnard.

Rather than target a tumor - suppressor gene directly, Ideker and team took the approach of identifying genetic interactions between a tumor suppressor gene and another gene, such that simultaneous disruption of both genes selectively kills cancer cells.

With the data provided by the FLI researchers, scientists around the world may now set targeted mutations to investigate the relevance of single genes for aging, in order to better understand regulating mechanisms and, eventually, develop new approaches for therapy or prevention of aging - related diseases.

This understanding of cancer's evolution has key implications for treatment, he noted, «In addition to pointing out that we will need to target driver genes that are mutated early in cancer, this evolutionary approach provides a method that accurately characterizes which genes are being mutated early and late.

His team's approach is based on gene therapy, where a «tame» virus is harnessed to transfer a gene into target cells in the recipient.

One general benefit of the RNAi approach, Zhang says, is that researchers can selectively target specific insect pests by targeting species - specific gene sequences; this avoids the blanket destruction of other insect species seen with many insecticides, he explains.

As the central nervous system is also a target of AAV vectors, Hélène Puccio and Patrick Aubourg's teams are investigating whether a similar approach using gene therapy could be as effective for the spinal cord and cerebellum as it is for the heart.

Experimental approaches such as gene therapy are also being investigated, but Dr. Rudnicki's research suggests that these approaches will have to be modified so that they target muscle stem cells as well as muscle fibres.

Her team integrates both computational and experimental approaches to explore how perturbations to these gene networks can alter morphology, both within a species and across the grasses, with the ultimate goal of defining targets for improving grain yield in cereals.

While circulating tumor DNA tests targeting a smaller set of cancer genes are already available for use in routine practice to guide care, by covering a much larger number of cancer genes, this high - intensity sequencing approach may enable development of future tests for early detection of cancer.

But so far, silencing genes using RNAi has not worked well in mammalian cells; although the approach has been successful in mouse embryos, double - stranded RNA shuts down synthesis of all proteins, not just the target gene, in other types of mammalian cells.

This common genetic heritage is a potential achilles heel of the metastases, however, many of these shared mutations are in tumor suppressor genes and our approach to therapeutically targeting these needs to be prioritised.

In an effort to combine the respective advantages of the AAV and CRISPR - based approaches, several groups have recently worked to enhance the efficiency of AAV - based gene editing via the introduction of a double strand break by using a targeted nuclease.

Utilizing genetic approaches such as «fine mapping» and «genome editing,» the researchers narrowed the region of the chromosome and targeted each gene to responsible for affecting the behavioral response to the drug.

First, unlike CRISPR, the AAV - based approach is only able to target a single allele of a single gene at once.

«This study suggests that targeting p27, GATA3 and POU4F3 may enhance the outcome of gene therapy and other approaches that aim to restart ATOH1 expression,» Zuo said.

However, like most gene transfer approaches a major concern with ZFN technology is the potential for oncogenesis due to off - target effects.

This study is good example of a precision medicine themed approach to showing how a mechanistic understanding of the differential gene expression can yield biomarkers that can be used for early detection of disease, as well as potential therapeutic targets.

His work centers on developing general, rational approaches to design precision medicines from genome sequences by targeting the RNA product of genes.

Through its population - based approach and three main business units providing disease - gene and drug target identification, database services and informatics tools, deCODE is turning raw genomics data into products and services for the healthcare industry.

This service will complement the shRNA core facility as many gene functions are masked or skewed due to the partiality, off - target, and transient nature of the knockdown approach.

This approach will allow you to insert luminescent or fluorescent reporter genes in specific target sites.

Also, a different targeting approach leading to the use of the native 3 ′ UTR and removal of the neo gene resulted in normalisation of XpdXPCS mRNA levels and viable homozygous XpdXPCS / XPCS (XPDG602D / G602D) animals [23].

More specifically, he has developed therapeutic approaches that target gene - control mechanisms in the stressed and failing heart, a process that has striking similarities to uncontrolled growth in cancers.

This approach allows to obtain models in shorter timelines and with reduced costs compared to gene targeting by homologous recombination in ES cells.

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.

Most therapeutic approaches ignore the dynamic molecular network of genes, targeting instead...

In an interdisciplinary approach, the team will first systematically identify genes that are required for this differentiation process - information which will then be used to investigate their molecular mode of action, their targets, and if and how they interact with each other.

We are using live imaging, transgenic reporters of osteoblast gene expression and targeted RNAseq approaches to identify the molecular regulators of progressive osteoblast differentiation during expansion of the skull.

Ensuring appropriate target gene selection and maintaining a diversified pre-clinical engine are key aspects of our strategic approach.

We have demonstrated that our liposomes can mediate in vivo target mRNA delivery as well as gene disruption using an entirely RNA - based approach.

Following a Forward Genetics approach, Fleming researchers identified a novel neurological mouse model caused by a functional mutation in the Slc25a46 gene, a new pathogenic target in a wide spectrum of human neurological diseases, including optic atrophy, Charcot - Marie - Tooth type 2, Leigh syndrome, progressive myoclonic ataxia and lethal congenital pontocerebellar hypoplasia.

Once these target genes are identified, he then uses a combination of gene therapy - and medication - based approaches to block the function of these genes and potentially halt the disease.

The approach to target validation utilizes RNAi, CRISPR, and ORF expression platforms to genetically perturb candidate target gene expression in relevant cancer cell lines, and then profile the resulting phenotypic changes with regard to their effect on various biochemical pathways.

«Our breakthrough findings in psoriasis underscore the unique power of our population approach for tackling the genetics of just about any common condition, and we hope to push this program forward to identify the gene involved as well as targets for the development of new therapies,» said Dr. Kari Stefansson, CEO of deCODE genetics.

Given the tailored inhibition of selected genes and the added precision brought by targeted delivery systems, RNAi - based therapies are thought to carry lower risk of failure than traditional approaches as the biological effects are more predictable.

The gene - targeting approach developed by Suzuki and his colleagues relies on the use of so - called helper - dependent adenoviral vector to deliver large mutation - free DNA molecules into cells.

This approach allows to obtain mouse models in shorter timelines and with reduced costs compared to gene targeting by homologous recombination in ES cells.

Their contributions to the yeast community include physical mapping methods, synthetic lethality screen approaches for identifying cross-species candidate genes as potential cancer drug targets, and a widely used set of vectors and yeast host strains that have been instrumental in work that has led to countless discoveries in recent decades.