#Gene therapy restores hearing in deaf mice Proof-of-principle study takes a step toward precision medicine for genetic hearing loss.
Using gene therapy, researchers at Boston Children Hospital and Harvard Medical school have restored hearing in mice with a genetic form of deafness.
Their work, published online July 8 by the journal Science Translational Medicine, could pave the way for gene therapy in people with hearing loss caused by genetic mutations. ur gene therapy protocol is not yet ready for clinical trialse need to tweak it a bit moreut in the not-too-distant
The researchers tested gene therapy in two types of mutant mice. One type had the TMC1 gene completely deleted
and is a good model for recessive TMC1 mutations in humans: Children with two mutant copies of TMC1 have profound hearing loss from a very young age, usually by around 2 years.
The other type of mouse, called Beethoven, has a specific TMC1 mutation change in a single amino acidnd is a good model for the dominant form of TMC1-related deafness.
In this form, less common than the recessive form, a single copy of the mutation causes children to gradually go deaf beginning around the age of 10 to 15 years.
or AAV1, together with a promoter genetic sequence that turns the gene on only in certain sensory cells of the inner ear known as hair cells.
In the recessive deafness model, gene therapy with TMC1 restored the ability of sensory hair cells to respond to soundroducing a measurable electrical currentnd also restored activity in the auditory portion of the brainstem.
loud tones. ice with TMC1 mutations will just sit there, but with gene therapy, they jump as high as a normal mouse,
says Holt. The force of their jump was measured by a plate on the floor underneath them;
In the dominant deafness model, gene therapy with a related gene, TMC2, was successful at the cellular and brain level,
and is already in use in human gene therapy trials for blindness, heart disease, muscular dystrophy and other conditions.
Holt hopes to partner with clinicians at Boston Children Department of Otolaryngology and elsewhere to start clinical trials of TMC1 gene therapy within 5 to 10 years. urrent therapies for profound hearing loss like that caused by the recessive
Holt believes that other forms of genetic deafness may also be amenable to the same gene therapy strategy.
000 live births. can envision patients with deafness having their genome sequenced and a tailored, precision medicine treatment injected into their ears to restore hearing,
a mutation in the TMC1 gene is sufficient to cause deafness. However, Holt study also showed that gene therapy with TMC2 could compensate for loss of a functional TMC1 gene,
restoring hearing in the recessive deafness model and partial hearing in the dominant deafness model. his is a great example of how the basic science can lead to clinical therapies,
says Holt. he implications of successful gene therapy are profound, and we are delighted to be associated with this study program,
such strains are only found at low levels within the human gut, according to Timothy Lu, an associate professor of biological engineering and of electrical engineering and computer science,
a professor of biological engineering at MIT. e wanted to work with strains like B. thetaiotaomicron that are present in many people in abundant levels,
The team developed a series of genetic parts that can be used to precisely program gene expression within the bacteria. sing these parts
we could engineer bacteria to detect multiple biomarkers, and only trigger a response when they are all present.
Tom Ellis, group leader of the Centre for Synthetic biology at Imperial College London, who was involved not in the research,
says the paper takes many of the best tools that have been developed for synthetic biology applications with E coli
or biosensors, in bacteria that are placed then in the gut, this paper stands out from the crowd by first engineering a member of the Bacteroides genus,
said Dr. Wen H. Shen, the study lead investigator and an assistant professor of cell biology in radiation oncology at Weill Cornell. ased on our research,
and if loss of PTEN could impact this central process of genome transmission to allow development and progression of cancer.
or when cells with unreplicated DNA rush into cell division prematurely to produce an abnormal number of chromosomes in a cell, a condition called aneuploidy.
70 percent of prostate cancers have PTEN mutation or deletion. Because of this, researchers are testing PTEN to see if it a marker of aggressive cancer
or harbor mutations in the gene are known to have poorer outcomes than patients with active PTEN,
and in particular, to benefit this subgroup of cancer patients carrying PTEN mutations. ource: Cornell Universit u
Based on these preclinical data, the HIV-1 version of this vaccine regimen is now being evaluated in an ongoing international clinical study sponsored by Crucell Holland BV
The remaining particles degrade easily after disposal because of their biocompatible lignin core, limiting the risk to the environment. eople have been interested in using silver nanoparticles for antimicrobial purposes,
INVISTA Professor of Chemical and Biomolecular engineering at NC State and the paper corresponding author. e show here an inexpensive and environmentally responsible method to make effective antimicrobials with biomaterial cores. he researchers used the nanoparticles
Ralstonia, a genus of bacteria containing numerous soil-borne pathogen species; and Staphylococcus epidermis, a bacterium that can cause harmful biofilms on plastics like catheters in the human body.
The nanoparticles were effective against all the bacteria. The method allows researchers the flexibility to change the nanoparticle recipe in order to target specific microbes.
the researchers used biochemical and biophysical cues to prompt stem cells to differentiate and self-organize into micron-scale cardiac tissue,
said Kevin Healy, a UC Berkeley professor of bioengineering, who is co-senior author of the study with Dr. Bruce Conklin,
a senior investigator at the Gladstone Institute of Cardiovascular disease and a professor of medical genetics and cellular and molecular pharmacology at UC San francisco. his technology could help us quickly screen for drugs likely to generate cardiac birth defects,
which are critical to the development of heart tissue. his spatial differentiation happens in biology naturally,
a UC Berkeley postdoctoral researcher in bioengineering. he confined geometric pattern provided biochemical and biophysical cues that directed cardiac differentiation and the formation of a beating microchamber.
according to new research published in Biomaterials. The authors of the study, from Osaka University in Japan, say their dissolvable patch the only vaccination system of its kind could make vaccination easier, safer and less painful.
and biology research because it can reveal how molecules stick together. It widely utilized in an eclectic collection of fields spanning anthropology, fuel science, physiology and meteorology.
an electrical engineer whose work often concerns the semiconductor industry. ut wee also excited for the huge number of people who can now use this technique in chemistry and biology.
Jae-Woong Jeong, Ph d.,a bioengineer formerly at the University of Illinois at Urbana-Champaign, worked with Jordan G. Mccall, Ph d.,a graduate student in the Bruchas lab,
as measured by real-time polymerase chain reaction. CONCLUSIONS: Bone marrow-derived MSC sheets improved cardiac function and attenuated LV remodeling in ICM without major complications,
a finding that dispels previous concerns that the genetic material would quickly degrade in rain and sunlight.
Geneticists have used the technique to silence specific genes examine what functions are lost and hence learn that gene purpose.
it could more easily detect smaller amounts of biomarkers, thus providing earlier diagnosis and prognosis of conditions such as cancer, said Dr. Andreas Holzinger,
she said. he only exotic component is the carbon nanotube aerogel sheet used for the fiber sheath.
to manufacture carbon nanotube aerogel sheets for diverse applications c
#New receptor for controlling blood pressure discovered High blood pressure is a primary risk factor in the development of many cardiovascular diseases.
#How to make chromosomes from DNA Researchers at the University of Tokyo have discovered a long-overlooked process important for converting a long, string-like DNA molecule into a chromosome.
This finding gives us a better understanding of the mechanism of how cells store safely genetic material, DNA.
Condensin recognizes unwound DNA segments produced by gene expression and restores them to double-stranded DNA.
This function proved to be a prerequisite for making chromosomes from DNA. DNA molecules are long,
string-like polymers storing the genetic information of life and, in a cell, are packed tightly into structures called chromosomes.
Formation of chromosomes in a dividing cell is required for faithful transmission of information in DNA to daughter cells.
The condensin complex is known to play an essential role in assembling chromosomes, but it remains unknown how condensin is involved in folding of DNA molecules.
Researchers at the University of Tokyo, including Assistant professor Takashi Sutani, Professor Katsuhiko Shirahige (Institute of Molecular and Cellular Biosciences) and Ph d student Toyonori Sakata (Graduate school of Agricultural and Life sciences), isolated from cells
and analyzed DNA segments to which condensin binds, and revealed that condensin is associated with single stranded-dna DNA (ssdna)
and were produced by gene expression (or transcription), and that ssdna amount was increased further in condensin-deficient cells.
They also discovered that chromosome segregation defects in mutant cells that showed lowered levels of condensin function were rescued largely by transcription inhibition.
that ssdna is detrimental to assembling chromosomes, and that condensin restores unwound ssdna segments to double-stranded DNA. t was believed widely that unwound DNA segments return spontaneously to canonical double-helical DNA,
It has demonstrated also for the first time that the presence of ssdna impedes chromosome organization, providing insight into the mechanism of chromosome formation,
says Assistant professor Sutani u
#Discovery: cells unwillingly help adenoviruses Various viruses claim many lives every day and cause other nonlethal infections that can lead to serious complications.
but they are used also in sciences adenoviruses are used widely in gene therapy. Scientists found out that cells unwillingly provide lipids,
which is surprising as lipids have important roles in biology, but these roles are difficult to identify The researchers have identified a connection between the formation of a membrane pore by the virus and a cellular repair mechanism.
New knowledge should also help with using adenoviruses in vaccination and gene therapy e
#Scientists discover first NA ambulanceu of T researchers have discovered how severely damaged DNA is transported within a cell
a Professor in the Faculty of medicine Department of Laboratory Medicine and Pathobiology. ee now discovered the DNA mbulanceand the road it takes. ekhail discovered this DNA ambulance,
because DNA contains the instructions for all our genetic information. While the repaired DNA can still replicate,
but at a great cost, said Mekhail. he cell has compromised a genome, but it stable
when our chromosomes break and are said misrepaired Durocher. his work teaches us that the location of the break within the cell nucleus has a big impact on the efficiency of repair. he implications of the research could extend to a large number of developmental
The examination of epigenomes requires mapping DNA interactions with a certain protein in the entire genome.
At one point, the study of in vivo genome-wide protein-DNA interactions and chromatin modifications required approximately 10 million cells for an individual test.
The latest breakthrough comes from Lu collaboration with Kai Tan at the University of Iowa, a systems biologist and associate professor of internal medicine.
and that are so short that the biomolecule structure is imaged before it is destroyed. This capability allowed the team to create the three-dimensional image of the arrestin-rhodopsin complex at an atomic level a much higher resolution than is possible with conventional X-ray technologyfemtosecond X-ray pulses are almost unfathomably brief.
researchers Nadia Zatsepin and Stella Lisova from the Department of physics as well as the graduate students Shibom Basu, Jesse Coe, Chelsie Conrad and Shatabdi Roy-Chowdhury from the Department of chemistry and Biochemistry,
assistant professor in the Department of chemistry and Biochemistry and member of the Center for Applied Structural Discovery. his study provides important clues about how we can improve human health
MIT is negotiating an exclusive license agreement with Lyndra, an early-stage biotechnology company developing novel oral drug-delivery systems,
scientists have devised a new strategy to precisely modify human T cells using the genome-editing system known as CRISPR/Cas9.
and inexpensively edit genetic information in virtually any organism. T cells, which circulate in the blood, are an obvious candidate for medical applications of the technology,
But in practice, editing T cell genomes with CRISPR/Cas9 has proved surprisingly difficult, said Alexander Marson, Phd, a UCSF Sandler Fellow,
said that the research is a significant step forward in bringing the power of CRISPR/Cas9 editing to human biology
and allows new genetic sequences to be inserted, has generally been introduced into cells using viruses or circular bits of DNA called plasmids.
(or nock in specific new sequences to correct mutations in T cells. As will be reported online in Proceedings of the National Academy of Sciences during the week of July 27
so there increasing clinical infrastructure that we could potentially piggyback on as we work out more details of genome editing,
#Scientists create functional liver cells from stem cells Major implications for liver biology and drug discovery The liver plays a critical role in human metabolism.
in research published on the cover of the July edition of Hepatology, scientists from the Hebrew University of Jerusalem Alexander Grass Center for Bioengineering report that they produced large amounts of functional liver cells from human
can detect the toxic effect of over a dozen drugs with greater than 97%accuracy. he implications for liver biology
said V. Reggie Edgerton, senior author of the research and a UCLA distinguished professor of integrative biology and physiology,
and Bioengineering (grants U01eb15521 and R01eb007615), the Christopher and Dana Reeve Foundation, the Walkabout Foundation and the Russian Scientific Fund. hese encouraging results provide continued evidence that spinal cord injury may no longer mean a lifelong sentence of paralysis
director of the National Institute of Biomedical Imaging and Bioengineering. he potential to offer a life-changing therapy to patients without requiring surgery would be a major advance;
It a wonderful example of the power that comes from combining advances in basic biological research with technological innovation. dgerton estimates that cost to patients of the new approach could be one-tenth the cost of treatment using the surgical epidural stimulator
and is director of the laboratory of movement physiology at Russia Pavlov Institute and a researcher in the UCLA department of integrative biology and physiology,
differing by specific changes in the genetic material of the leukemia cells, but also by their response to therapies.
which occurs due to breakage and aberrant fusion of genetic material in the tumour cells, resulting in the formation of a new oncogenic protein encoded by parts of the genes TCF3 and HLF, respectively (TCF3-HLF-positive leukemia cells).
An international group of clinicians and scientists from different universities and research institutions, among them the Berlin-based Max Planck Institute for Molecular genetics (Department of Vertebrate Genomics, Hans Lehrach, group
With the aim of identifying therapeutic entry points for this incurable form of ALL THE consortium team decoded both the genome and the transcriptome of the cancer cells,
Using sophisticated bioinformatics methods, the team found genetic aberrations in addition to the known translocation, and deciphered the so-called expression profile of the cancer cells by means of RNASEQ, a focus of research in Marie-Laure Yaspo group in Berlin.
and an altered gene expression program leads to a reprogramming of leukemic cells to an early, stem-cell like, developmental stage,
. director of the National Institute of Biomedical Imaging and Bioengineering at NIH. he potential to offer a life-changing therapy to patients without requiring surgery would be a major advance;
It a wonderful example of the power that comes from combining advances in basic biological research with technological innovation. he study was conducted by a team of researchers at the University of California, Los angeles;
The team was led by V. Reggie Edgerton, Ph d.,a distinguished professor of integrative biology and physiology at UCLA and Yury Gerasimenko, Ph d.,director of the laboratory of movement physiology at Pavlov Institute and a researcher
in UCLA Department of Integrative biology and Physiology. They reported their results in the Journal of Neurotrauma.
but the researchers expect expenses to drop over time (as has been the case with genome sequencing).
called Ribo-T, was created in the laboratories of Alexander Mankin, director of the UIC College of Pharmacy Center for Biomolecular Sciences,
The engineered ribosome may enable the production of new drugs and next-generation biomaterials and lead to a better understanding of how ribosomes function.
called Ribo-T, was created in the laboratories of Alexander Mankin, director of the UIC College of Pharmacy Center for Biomolecular Sciences,
and Northwestern Michael Jewett, assistant professor of chemical and biological engineering. The human-made ribosome may be able to be manipulated in the laboratory to do things natural ribosomes cannot do.
or producing designer therapeutics and perhaps one day even non-biological polymers. No one has developed ever something of this nature. e felt like there was a small very small chance Ribo-T could work
Jewett. ur new protein-making factory holds promise to expand the genetic code in a unique and transformative way, providing exciting opportunities for synthetic biology and biomolecular engineering,
a professor of chemistry and chemical biology at Harvard university. Existing implantable electrodes are too large and rigid,
and biocompatible materials (see ireless Micro LEDS Control Mouse Behavior, but none have overcome this problem,
The new nanoelectronic eshstructure that Lieber group has designed is much more like the biological tissue it is meant to interface with,
and Canadian researchers has managed to successfully sequence the full genome of a living organism using a machine the size of a smartphone called the Minion.
"It's amazing,"added Luiz Ozaki from the Center from the Study of Biological Complexity,
and get a genetic sequence right there.""The drawbacks identified so far revolve around accuracy-the data it produces is currently less accurate than the data produced by a larger machine,
meaning that strong bioinformatics tools are needed to correct errors. That was the focus of Simpson's paper:"
and develop ways to reconstruct complete genomes off this tiny sequencer, "he said. Once proven, it's hoped that the device could be used to sequence tumour genomes,
giving the option of more personalised diagnosis and treatment to cancer patients.""It's not ready for prime time yet,
"said Gregory Buck, also from the Center from the Study of Biological Complexity.""We want to see it work,
#Scientists are turning your gut cells into computers A team of biological engineers at the Massachusetts institute of technology are turning the cells in our guts into computers.
"We hope our work provides inspiration for more research into the development of materials that mimic biological organisms."
and look at their genomes we can recognize many equivalences, said molecular biologist Edward Marcotte of the University of Texas at Austin who led the new study. n fact,
there are thousands of genes shared between humans and yeast. These shared genes may appear similar,
For example, the sets of genes involved in sterol biosynthesis and in the proteasome complexhe cell garbage disposalere almost all replaceable,
#Optogenetics Meets CRISPR The CRISPR gene-editing system just got even better: a new light-activated Cas9 nuclease could offer researchers greater spatial and temporal control over the RNA-guided nuclease activity,
according to a study published today (June 15) in Nature Biotechnology. his is an effective new system for extremely precise control of gene editing via light, Paul Knoepfler, a stem cell biologist at the University of California, Davis,
and control of genetic modification is an important advance, he added, noting that his is one of many such efforts.
using its Magnet proteins to create a photoactivatable Cas9 nuclease (pacas9) for light-controlled genome editing. he existing Cas9 does not allow to modify genome of a small subset of cells in tissue, such as neurons in the brain,
We have been interested in the development of a powerful tool that enables spatial and temporal control of genome editing.
Sato said. he new study makes use of the split Cas9 architecture to enable light-activated genome editing,
Sato group now plans to expand the colors of light that can activate the pacas9 nuclease o make genome editing more flexible
hotoactivatable CRISPR-Cas9 for optogenetic genome editing, Nature Biotechnology, doi: 10.1038/nbt. 3245,2015 d
#Tasty Visuals To help those with visual impairments make out the shape and movement of objects, a company has developed a device that takes visual information
said Shu Yang, a professor in Penn Engineering departments of Materials science and engineering and Chemical and Biomolecular engineering.
an observation that could have important implications with the increased attention to immune-system-based cancer therapies. e are only at the beginning of our quest to understand the role and biology of CTC clusters,
from Strathclyde Department of Biomedical engineering, has developed the innovative system, known as Majicast, to manufacture lower limb prosthetic sockets that fit prostheses securely to patientsresidual limbs.
Three-dimensional structures in materials and biological samples can be investigated using X-ray tomography, which is done by recording images layer-by-layer
our biological and technological distinctiveness will be added to our own. Resistance is futile. But in reality, Clune says,
ntil now we thought that spider silk was the strongest biological material because of its super-strength
when designing new structures but with so many biological structures to consider, it can take time to discover which may be useful.
#White house unveils $215 million plan to develop patient-specific medical treatments The White house unveiled a"Precision Medicine Initiative"today a $215 million investment that will go toward building a database containing genetic information
The investment, one that part of President Obama 2016 budget proposal, will also go toward pioneering new models for patient-specific medical research research that caters to a specific patient needs based on their genetics instead of relying on a one-size-fits-all
or more volunteers"whose genetic information will be stored in a series of databases. About $70 million will go to the National Cancer Institute, a subsection of the NIH,
so it can increase its efforts to identify genetic mutations that contribute to a person's cancer risk.
The Genetic information Nondiscrimination Act was signed into law in 2008 to prevent genetic discrimination from happening,
says Michael Eisen, a geneticist at the University of California, Berkeley.""I worry that this $200 million will ultimately come from basic research funding,
a computational biology researcher and co-author on the paper. And when the team removed the Nix gene from male mosquitoes,
a professor of biochemistry in the College of Agriculture and Life sciences. There's so much information to sift through that it was simply too daunting of a numbers game.
"The mosquito genome is like an unfinished puzzle""Jake and his students found a way to look in those pieces
what's referred to as the"black hole"of the genome regions full of duplicate information that are extremely hard to sequence.
and eventually exploit this male-determining factor by using transgenics, essentially changing this specific gene in mosquitoes on a deep enough cellular level that they pass it on to any offspring."
researchers detail how sequencing genetic information in sperm can show whether a man is infertile. The scientists looked at the sperm RNA,
which carries essential genetic information. But around 20 years ago, Krawetz and his research team discovered that sperm also house RNA
Since sperm RNA correspond to genes in a child genome, the researchers may be able to study these sequences to see
synthetic biologist at Northwestern University and a co-author of the study. These ribosomes could be tailored to only make a certain type of molecule,
Farren Isaacs, a cellular biologist at Yale university, agrees. The study is"a key advance,"that"sets the stage for the production of entirely new classes of exotic molecules."
a molecular biologist at the University of Illinois at Chicago and a co-author of the study."
using a zero-knowledge proof to protect sensitive biometric data. More importantly, a system that's compatible with FIDO can accept a login from any FIDO-certified device,
letting users authenticate through a combination fingerprint reader and biometric wristband. Samsung's secure identification framework was certified also,
"The technology has been developed by a team of bioengineers led by Ozgur Sahin at Columbia University by making use of the property of bacterial spores,
Finette, 58, is a professor of pediatrics, microbiology and molecular genetics at the University of Vermont College of Medicine and director of the Global Health and Humanitarian Opportunity Program.
Ed Boyden, an associate professor of biological engineering and brain and cognitive sciences at MIT, and the lead author of the study,
move around, do things that biological muscles do added Solomon. If the technology of microbots attains success on a large scale,
which initially focused on filtering biological contaminants from groundwater to make it potable. She joined MIT Mechanical engineering assistant professor Amos Winter's laboratory in 2012,
The ultraviolet light from the sun rays are used to kill biological contaminants in the water. While similar desalination technologies have already been discovered,
#After Celgene Deal, Juno Revs Armored CAR For Ovarian Cancer Trial Juno Therapeutics has built remarkable momentumncluding last week much-dissected $1 billion deal with Celgenepon
< Back - Next >
Overtext Web Module V3.0 Alpha
Copyright Semantic-Knowledge, 1994-2011