#New system for human genome editing has potential to increase power and precision of genome engineering A team including the scientist who first harnessed the CRISPR-Cas9 system for mammalian genome editing has identified now a different CRISPR system with the potential for even simpler and more precise genome engineering.
In a study published today in Cell, Feng Zhang and his colleagues at the Broad Institute of MIT and Harvard and the Mcgovern Institute for Brain Research at MIT,
and demonstrate that it can be engineered to edit the genomes of human cells. his has dramatic potential to advance genetic engineering,
but also shows that Cpf1 can be harnessed for human genome editing and has remarkable and powerful features.
The Cpf1 system represents a new generation of genome editing technology. CRISPR sequences were described first in 1987
The application of the CRISPR-Cas9 system for mammalian genome editing was reported first in 2013, by Zhang and separately by George Church at Harvard university.
This could be an advantage in targeting some genomes, such as in the malaria parasite as well as in humans. he unexpected properties of Cpf1 and more precise editing open the door to all sorts of applications,
with other enzymes that may be repurposed for further genome editing advances. e
#Bubble, bubble, at the flick of a switch Boiling water, with its commotion of bubbles that rise from a surface as water comes to a boil,
Researcher finds keys to genome integrity Maintaining the stability and the correct sequence of our genetic information is vital to the accurate transmission of our genetic code.
However, in the course of replicating, our DNA frequently runs into roadblocks, arising from both internal and external sources,
that threaten the fidelity of our genetic information. The accurate processing of these roadblocks is paramount to genome integrity.
Defects in this process can lead to cancer, genetic problems and premature aging. In a research paper published in the Journal of Cell biology, Alessandro Vindigni, Ph d.,professor in the Edward A. Doisy Department of Biochemistry and Molecular biology at Saint louis University
Improper repair of DNA lesions can lead to mutations, abnormal chromosome structures, or loss of genetic information that in turn can cause premature aging, cancer, and genetic abnormalities.
Depending on the degree of genome instability these alterations will determine whether a cell survives, goes into a growth-arrest state, or dies.
If the cell's replication machinery collides with the lesion, a strand break can occur."
The technique will also allow researchers to examine gene expression in individual cells without removing them from tissue,
#Novel online bioinformatics tool significantly reduces time of multiple genome analysis UK research collaboration develops a new bioinformatics pipeline that enables automated primer design for multiple genome species
A key aspect of this is utilising breakthroughs in genomics research to guide the selection of the individuals to incorporate in breeding schemes.
Crop breeding programmes can make use of this genetic information to ensure that the preferred trait is inherited by future crop yields,
However, the majority of tools for the analysis of DNA are designed for diploid organisms, such as humans, with one set of chromosomes,
. which has multiple sets of chromosomes. Scientists from The Genome Analysis Centre (TGAC) and John Innes Centre have developed a bioinformatics pipeline
Polymarker that facilitates the design of genomic specific primers for polyploid species. Once identified, these primers can be used to ascertain
whether or not an individual organism has associated the genetic variation with a given trait. As an open access tool, researchers and crop breeders can submit their own data to Polymarker
and the online tool will return suggested design primers to identify genetic variations that tag vital traits in their crop samples,
and applied in a research project where it identified genetic markers that signal resistance to the wheat yellow rust pathogen (Puccinia striiformis f. sp. tritici).
In one new software tool, we have applied expertise in advanced algorithm development, knowledge on genetics and principles of genome architecture."
the researchers first placed the 5clc lesion at a specific site within the genome of a bacterial virus. They then replicated the virus within the cell.
the researchers replicated the genome containing the lesion with a variety of different types of polymerase,
whether genetic material has been delivered successfully to cells that scientists are trying to genetically alter. Cells that did not receive the new gene could be induced to undergo cell death,
or to study the 3-D structure of normal chromosomes by testing whether two genes located far from each other on a chromosome fold in such a way that they end up next to each other,
the researchers say
#Bioadhesive Nanoparticles Help Protect Your Skin From the Sun Dermatologists from Yale university have developed a new sunscreen made with bioadhesive nanoparticles that doesn penetrate the skin,
But pediatricians typically can scan an infant entire genome and analyze it for clues quickly enough to make a difference in the baby treatment.
and analyze the entire genome of a critically ill infant to find a diagnosis that can significantly alter the course of treatment.
In a new study published in Genome Medicine, pediatricians explained how hardware and software specialized for genetic analysis can provide such fast and lifesaving information.
The key piece of technology: A processor from the company Edico Genome that designed to handle the big data of genetics.
Lead researcher Stephen Kingsmore, a pediatrician and genomics expert at Children Mercy Hospital in Kansas city, explains that doctors typically run targeted genetic tests for specific diseases
if they have a good guess about what wrong with an infant. Such tests check a few specific spots on the genome,
looking for disease-causing mutations. But with more than 8000 possible genetic diseases, such tests eren really relevant to clinical care, he tells IEEE Spectrum.
Whole-genome sequencing is a different matter entirely. These scans check for mutations at each of the 3. 2 billion locations on the human genome.
Remarkably while it took $3 billion to sequence the first human genome, it can now be done for about $1000 a pop.
That cheap enough to make economic sense in medical emergencies, like those encountered in a neonatal intensive care unit.
Kingsmore 26-hour diagnostic pipeline starts with the machines that do the brute-force work of sorting through an individual baby genome.
came from the technology that analyzed each infant genome. That task is like taking the completed 3-billion-piece jigsaw puzzle
Using Edico Genome DRAGEN processor, the researchers got this step down from 15 hours to 40 minutes.
the researchers had a file listing all the mutations in a sick baby genome. After that, Kingsmore team used in-house software to search through the mutations for those associated with a disease that matched the baby symptoms.
In a prior study, Kingsmore team used whole-genome sequencing for 35 sick infants, and diagnosed a genetic disease in 20 of those babies.
In other cases, the genome scan allowed doctors to rule out diseases, which Kingsmore says can be equally valuable. octor always worry:
says Pieter van Rooyen, CEO of Edico Genome. The data comes from the sequencing machine in a particular file format,
every infant born in the developed world will have sequenced its genome in the hospital.?It just a matter of time before clinical genomics will be with us everywhere,
he says. t prudent to have the infrastructure ready. He envisions the DRAGEN processor outputting its analysis directly into a patient electronic medical record,
from genome sequencing to diagnosis, doesn alarm Kingsmore. In fact, he thinks it will be necessary if we want to make use of today best genetic technologies:
Most tech innovation in agriculture was concentrated narrowly in biotechnology and seed genetics, and both investment and innovation was limited to players with close ties to the ag sector.
Outside of seed genetics and crop inputs, most other Agtech was bundled typically with Cleantech. Then, in 2013, there was a shift.
Each of the robots carries a"genome""made up of different genes, like humans do.
"Our findings provide a genetic marker that could be used to identify those RA patients who require more aggressive treatments
"but are, in fact, important in regulating gene expression.""Our results provide novel insights into the regulation of early embryonic development in human,"added Outi Hovatta, professor at Karolinska Institutet's department of clinical science, intervention and technology.
The study is published in the open-access journal Genome Biology y
#Researchers identify many molecular machines critical for species development A new study shows a common molecular tool kit shared by organisms across the tree of life
when they published a paper describing attempts to edit the genomes of human embryos. Led by Junjiu Huang of Sun yat-sen University, Guangzhou,
when scientists found a strange thing in bacteria genomes. There were these repeating sequences with no known use.
Genetics was still developing and the scientists thought no more of this. Sometime later it was found that these CRISPR sequences were used by bacteria to ward off predatory viruses. The mechanism was unravelled
and manipulated for performing cut-andpaste functions on genomes. And the control was fantastic. You could precisely snip off a bit of DNA from a gene
or replace genetic material in a cell or organism.""What this means is: first, a piece of RNA is created for unzipping a DNA strand at the target site;
Its CEO Katrine Bosley said they are working to translate the promise of CRISPRCAS9 genome editing technology into a new class of medicines to treat serious, genetically driven diseases."
The new method-which uses the same type of waves used in medical sonograms-may have advantages over the light-based approach-known as optogenetics-particularly
In optogenetics, researchers add light-sensitive channel proteins to neurons they wish to study. By shining a focused laser on the cells,
I think we have shot a better with noninvasive sonogenetics approaches than with optogenetics, "he emphasised in a paper appeared in the journal Nature Communications.
The current gold standard for Ebola virus detection relies on a method called polymerase chain reaction (PCR) to amplify the virus's genetic material for detection.
In a series of experiments in breast cancer cell lines and transgenic mice that develop breast cancer as adults,
as release systems of biologically active species, cell encapsulation in silica porous materials, mesoporous materials for gene therapy and transfection, organic-inorganic hybrid materials.
in which progerin is linked to telomeresepetitive DNA sequences that protect the ends of human chromosomes
and gene expression differences induced by progerin. However, telomerase could not rescue the loss of heterochromatin.
transmit and express genetic information. Scientists are currently investigating ways of using nucleic acids to reprogram cells to carry out different functions.
"This is a quantum leap compared to existing technologies for the delivery of genetic material to cells and tissues,"said Ennio Tasciotti, Co-Chair, Department of Nanomedicine at Houston Methodist Research Institute and co-corresponding author of the paper."
#Super-Resolution Microscopy Helps Visualise and Count the Smallest Units in the Genome Now, for the first time,
which, packaged together, form our genome. This study was possible thanks to the use of super-resolution microscopy,
In combination with innovative quantitative approaches and numerical simulations, they were also able to define the genome architecture at the nanoscale.
Even though all the cells in our body have the same genetic information, they are not expressing all the genes at the same time.
or less accessible to the molecule that reads the genome: the RNA polymerase. Depending on the specialisation of the cells,
#Structured Illumination Microscopy and SPA Help Study SPB Duplication in Living Yeast Cells Cellular mitosis depends in part on small organelles that extend spindles to pull apart chromosome pairs.
One of the most critical biological and medical tools available today, it lies at the core of genome analysis. Reading the exact make-up of genes,
"and are strung together in various combinations that contain the cell's genetic information, such as genes. Essentially
The current gold standard for Ebola virus detection relies on a method called polymerase chain reaction (PCR) to amplify the virus's genetic material for detection.
Professor Lithgow and his team used a novel technology that enables the systematic expansion of the genetic codes of living organisms to include unnatural amino acids beyond the common twenty.
Elevations in the two examined gene markers of liver toxicity were detected then accurately and consistent with previously known changes, indicating that lab in a needle is an appropriate diagnostic option."
. who is a pioneer in the converging fields of synthetic biology, metabolic engineering, and genetics. Church is the Robert Winthrop Professor of Genetics at Harvard Medical school and Professor of Health Sciences and Technology at Harvard and MIT.
Critical to this process of metabolically engineering microbes is the use of biosensors. Made of a biological component-such as a fluorescent protein
whether genetic material has been delivered successfully to cells that scientists are trying to genetically alter. Cells that did not receive the new gene could be induced to undergo cell death,
or to study the 3-D structure of normal chromosomes by testing whether two genes located far from each other on a chromosome fold in such a way that they end up next to each other,
the researchers say
#Liquid crystals Detect Protein Fibers Associated with Development of Neurodegenerative Diseases Liquid crystals are familiar to most of us as the somewhat humdrum stuff used to make computer displays and TVS.
the researchers argue that their new method for controlling brain cells could improve on"optogenetics","a technique that uses light rather than sound.
Consequently, using optogenetics to control brain circuits in a mammal currently requires a fibre-optic implant.
#New age of genome editing could lead to cure for sickle cell anemia Australia researchers have shown that changing just a single letter of the DNA of human red blood cells in the laboratory increases their production of oxygen-carrying haemoglobin-a world-first
The new genome editing technique, in which a beneficial, naturally-occurring genetic mutation is introduced into cells,
"An exciting new age of genome editing is beginning, now that single genes within our vast genome can be precisely cut and repaired,
"says study leader, Dean of Science at UNSW, Professor Merlin Crossley.""Our laboratory study provides a proof of concept that changing just one letter of DNA in a gene could alleviate the symptoms of sickle cell anaemia and thalassaemia-inherited diseases in
"Because the good genetic variation we introduced already exists in nature, this approach should be effective and safe.
The researchers introduced this single-letter mutation into human red blood cells using genome-editing proteins known as TALENS,
When our genome editing protein cuts the DNA, the cell quickly replaces it with the donor DNA that we have provided also."
If the genome-editing technique is shown to work effectively in blood stem cells and be safe,
such as conventional gene therapy, in which viruses are used to ferry healthy genes into a cell to replace the defective ones.
and reform into spindles that are used by the dividing cell to move chromosomes. For chromosome migration to occur,
the microtubules attached to them must disassemble, carrying the chromosomes in the process. The dynamic instability that makes it possible for microtubules to transition from a rigid polymerized
or"assembled"nucleotide state to a flexible depolymerized or"disassembled"nucleotide state is driven by guanosine triphosphate (GTP) hydrolysis in the microtubule lattice."
years in advance has been developed in research published in the open access journal Genome Biology. The study aimed to define a set of genes associated with'healthy ageing'in 65 year olds.
New CRISPR-Cas9 strategy edits genes 2 ways The CRISPR-Cas9 system has been in the limelight mainly as a revolutionary genome engineering tool used to modify specific gene sequences within the vast sea of an organism
genome engineering and gene regulation, are initiated with a common step: the Cas9 protein is recruited to targeted genes by the so-called matching sequences of"GUIDE RNA"that help Cas9 latch on to specific sequences of DNA in a given genome.
But until now, genome engineering and gene regulation required different variants of the Cas9 protein; while the former task hinges on Cas9's innate DNA-cleaving activity,
the latter has been achieved by engineered Cas9 variants that have had their DNA-cleaving"fangs"removed, but still retain their ability to latch onto a specific genomic target.
These latter Cas9 variants are fused commonly with proteins that regulate gene expression. Now, using a new approach developed by researchers led by George Church, Ph d.,of Harvard and Ron Weiss, Ph d.,of the Massachusetts institute of technology,
Church is Core Faculty member at Harvard's Wyss Institute for Biologically Inspired Engineering, Robert Winthrop Professor of Genetics at Harvard Medical school and Professor of Health Sciences and Technology at Harvard and MIT,
we can now for the first time toggle a single protein to gain direct control over both, gene sequences and gene expression,
or mechanical stress on the chromosomes into which our genetic material is packaged. To make sure cells stay alive
For instance, different regions of a chromosome can fuse together, causing genes to rearrange themselves --and such chromosome fusions can lead to diseases such as cancer.
To learn more about the process, Kapoor, Kleiner and their colleagues zeroed in on the sites in chromosomes where DNA repair happens.
Specifically they focused on a single histone, a type of protein that DNA wraps around to make up chromosomes.
This histone, H2ax, is known to be involved in DNA repair. Immediately after DNA damage occurs, H2ax gets a mark--it becomes tagged with a chemical moiety known as a phosphate.
'Genome editing in human T-cells has been a notable challenge for the field, 'said lead author Dr Alexander Marson from the University of California, San francisco.'
'We have now been able to cut as well as paste pieces of the genome into human T-cells for the first time to our knowledge,
The Stanford sleuths analyzed a number of publicly available data sets containing results of studies that had assessed activity levels for the entire human genome in sepsis cases,
Patients with Down syndrome have three copies of chromosome 21 in their cells. Theye also 20 times more likely to contract childhood B-cell acute lymphoblastic leukemia than the general population,
making that chromosome an important avenue for researching the genetic basis of the cancer. major goal of my laboratory is to identify the specific gene or genes on chromosome 21 responsible for the increased incidence of leukemia in this population,
. and Lora S. Lurie Professor in Medicine-Hematology/Oncology and Biochemistry and Molecular genetics, and a member of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.
In previous work, Crispino and colleagues found that a gene on chromosome 21 called DYRK1A contributes to the development of leukemia.
Because they have extra copies of chromosome 21, children with Down syndrome have more DYRK1A than usual. his finding is exciting to us
#Degenerating Neurons Respond to Gene therapy Treatment for Alzheimer Disease Degenerating neurons in patients with Alzheimer disease (AD) measurably responded to an experimental gene therapy in
The gene therapy approach has progressed since to phase II trials at multiple test sites. Results have not yet been released.
'but are in fact important in regulating gene expression. In the current study, the researchers show that the newly identified genes can interact with the'junk DNA,
Jewett and his colleagues combined state-of-the-art genome engineering tools and engineered biological artsinto a lug-and-playprotein expression platform that is cell-free.
#SAM-Screener Epigenetic Methylation Test The United states Patent and Trademark Office has issued a patent to Cayman Chemical
These epigenetic modifications regulate gene expression, impacting normal growth as well as cancer and disease. Dysregulation of histone methylation patterns is observed in a variety of human cancers, inflammation,
Custom assay development to adapt this assay format to other HMTS is also available through Cayman epigenetic screening
stopped brain cancer in rats by delivering gene therapy through nanoparticles. The nanoparticles deliver genes for an enzyme that converts a prodrug called ganciclovir into a glioma cell killer.
As in cystic fibrosis, a current delivery method of gene therapy relies on using a virus, which can pose significant safety risks.
People with Down syndrome have an extra copy of the 21st chromosome where the production gene for the beta amyloid protein resides.
and Disassemble On Command Scientists have deciphered the genetic code that instructs proteins to either self-assemble
Because the laboratory identified the genetic sequences that encode this behavior they were able to point out a long list of human proteins that likely exhibit it. his paper shows the incredible richness of peptide sequences that already have this very simple switch,
whether genetic material has been delivered successfully to cells that scientists are trying to genetically alter. Cells that did not receive the new gene could be induced to undergo cell death,
or to study the 3-D structure of normal chromosomes by testing whether two genes located far from each other on a chromosome fold in such a way that they end up next to each other,
the researchers say
#Researchers Discover Method to Measure Stiffness of Arteries in the Brain UCLA researchers have discovered a noninvasive method to measure vascular compliance,
They identified gene expression profiles typical of different types of neurons newborn neural progenitors and radial glia,
of which exactly three are bound to the genetic material something Drennan said surprised her. hat the best part about science,
A team from Oxford university's Department of Physiology, Anatomy and Genetics led by Dr. Deborah Goberdhan worked with cancer doctor and researcher, Professor Adrian Harris,
said study author Rusty Gage, a professor in the Salk Institute Laboratory of Genetics. Researchers believe this technique will be very helpful to scientists studying age-related diseases
However, this technique did not guarantee cells with epigenetic signatures of older cells. This made it difficult to study the aging of the human brain
After, they compared the patterns of gene expression in the resulting neurons with cells taken from autopsied brains.
Different patterns of gene expression were recognizable using the direct conversion method, depending on the age of the person they were created from. he neurons we derived showed differences depending on donor age,
said Mertens. nd they actually show changes in gene expression that have been implicated previously in brain aging.
another form of muscle disease, exon skipping coaxes cells to kipover abnormal sections of the genetic code,
the molecules that function to regulate gene expression that are necessary to make the treatment. e are thrilled to be able to continue development of this promising treatment technique,
Canada and The netherlands, including a team from Oxford Wellcome Trust Centre for Human genetics have carried out a series of experiments to assess the accuracy
sequencing the genome of a laboratory strain of E coli (Escherichia coli K-12). Working to a single, shared protocol, the consortium produced 20 data sets with enough results to be able to quantify the data yield, quality,
more expensive devices so-called standard short-read technologies other researchers have shown that theye of high enough quality to infer full-length genomes from scratch, for the E coli bacterium, Influenza virus,
and the Saccharomyces cerevisiae yeast genomes. The researchers of this study point out, though, that there is work still to be done, to improve the reproducible delivery of molecules into the device and the clarity of the software it uses.
and Senior Analyst in Microbial Genomics at the Wellcome Trust Centre for Human genetics. using the Minion in a project with secondary-school students in Oxford,
MSU associate professor of microbiology and molecular genetics and co-author of the study.""We now know that ERMANI is an essential key,
"said first author Mackenzie Lind, a doctoral candidate at the Virginia Institute for Psychiatric and Behavioral Genetics at Virginia Commonwealth University in Richmond."
and we turn off the genetic information and the demographic and clinical information, and we see that with combined information,
This included data on genetic markers known as single-nucleotide polymorphisms; demographic data, such as subject age, gender, marital status, and education level;
and say that the original motivation was curiosity about how much of anatomy we could predict from genetics
borrowing tools from the developing field of optogenetics, which so far has been used mainly in brain science.
'Optogenetics uses genetic modification to alter cells so that they can be activated by light. Until now, it has mainly been used to activate individual cells
'A protein called channelrhodopsin was delivered to heart cells using gene therapy techniques so that they could be controlled by light.
However, as gene therapy moves into the clinic and with miniaturization of optical devices, use of this all-optical technology may become possible.
the gene-editing tool known as Crispr is on the brink of revolutionizing the field of genetics internationally.
#Gluten-free wheat quest undertaken by farmers Kansas farmers are paying for genetic research to figure out exactly why some people struggle to digest wheat.
but our excuse for not knowing anything about is that the genome for S. ovata was sequenced only a couple of years ago.
The 2013 genome announcement launched right into the good stuff by noting that. ovata uses N-methyl compounds
and diversification of the species.'For each robot child, there is a unique'genome'made up of a combination of between one and five different genes,
and'crossover',where a new genome is formed by merging genes from two individuals. In order for the'mother'to determine which'children'were the fittest
The geneticists have applied for a unique licence from the Government fertility watchdog to carry out the procedure using a gene manipulation technique called Crispr/Cas9.
If granted, it will be only the second occasion where the chromosome of human embryos have been modified.
Editing genetic codes to remove inherited disease has already been shown to work on mice. However most geneticists believe the procedure is not ready to be carried out on human embryos-after the Chinese research led to unwanted mutations in DNA.
The application for a licence to perform enome editingon human embryos has been made by Dr Kathy Niakan, of the Francis Crick Institute, London.
She emphasised that there would be o GM babiesbecause the team simply wants to understand the genetics of early embryonic development.
In 2008 the UK laws on IVF was changed to allow genetic manipulation of embryos that are less than two weeks old for research purposes,
of chromosomes passed on to future generations. But it continues to attract controversy as critics fear DNA alterations could create unforeseen health problems that would be passed down from generation to generation of the population.
'Dr David King, director of Human genetics Alert, a campaign group which opposes genetically-modified babies,
'Genome editing of embryos for use in treatment is illegal.''It has been permissible in research since 2009,
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