We are particularly interested in how
cells use gene networks to make robust decisions even in the presence of significant fluctuations in gene expression.
Next, the scientists removed the Duchenne mutations in the iPS
cells using a gene editing platform they developed that uses the CRISPR / Cas9 technology.
Our cells use genes as recipes to build proteins — little molecular machines that do useful stuff in cells.
This work, published in «Blood», was carried out by the CNIO Telomeres and Telomerase Group The treatment is based on the transport of the telomerase gene to the bone marrow
cells using gene therapy, a completely new strategy in the treatment of aplastic anaemia
Not exact matches
Birds, for example, can regenerate hair
cells — and lately researchers have found a few similar effects in mammals that
used gene and drug therapy.
Gene therapy, for example, frequently involves using a virus to deliver a gene to the cells that scientists are target
Gene therapy, for example, frequently involves
using a virus to deliver a
gene to the cells that scientists are target
gene to the
cells that scientists are targeting.
That's the path pursued by GenVec, which wants to be the Rogaine of the ears,
using gene therapy to regrow damaged hair
cells.
CRISPR
gene - editing has already been vaunted as a tool that could eventually be
used to tackle everything from HIV / AIDS to sickle
cell disease to a variety of other disorders.
Using the
gene - editing tool CRISPR - Cas9 to turn off certain
genes in a mouse zygote as well as other new techniques to enrich the pluripotent stem
cells of a rat, the group managed to grow various rat organs (a pancreas, heart, and eyes) in a mouse embryo.
Indeed, because eggs are large
cells that are relatively easy to manipulate, they are one of the favored
cell types
used by biologists to express foreign
genes and to test
gene function.
The OAR proposal
uses a variation of therapeutic cloning called altered nuclear transfer (ANT) in which the nucleus of a donor
cell (a skin
cell, for example), containing the 30,000
genes of the genetic code, is altered in such a way that it produces an epigenetic factor, a protein called nanog.
One of the key caveats at the time, however, was that the technique required the
use of a virus to introduce several
genes into the skin (or other)
cell, and these would remain in the
cell, and so might contaminate the resulting stem
cell or create cancer risks.
During normal development, epigenetic programming restricts both the
genes a
cell can
use and the
cell's developmental potency.
They include going after the damage to
cells done by free radicals, making
use of hormone therapy, or caloric restrictions, or vitamin supplements, or, most dramatically, healthy
gene selection through pre-implantation genetic diagnosis and even repairing the entire human genome.
iPS
cells tend to age prematurely and die; they are also created with cancer - causing
genes, which could make them dangerous to
use therapeutically.
Where is the clear line in a progression from (1)
using animal insulin to treat diabetes, to (2)
using gene remodeling techniques to grow insulin in a host bacterium that will reproduce rapidly and from which a plentiful supply of insulin can be harvested, to (3) genetic surgery to replace the defective
gene in a person diagnosed as diabetic, to (4) genetic surgery immediately after fertilization in order to replace the defective
gene and alter the germ
cells which would otherwise have transmitted the disease to one's offspring?
Though there have been many strides made towards ending the HIV / AIDS epidemic, such as the recent breakthrough of scientists
using gene editing to remove HIV from the genome of T -
cells, there is still much work to be done with over 1.2 million in the United States living with the disease.
«If we could
use gene editing to remove the sequences in an embryo that cause sickle
cell disease or cystic fibrosis, I would say not only that we may do so, but in the case of such severe diseases, we have a moral obligation to do so.»
Yoshinori Ohsumi, the most recent prizewinner,
used baker's yeast to identify
genes crucial in autophagy, the process by which
cells recycle their components.
Using viral
gene insertion and regulatory proteins, researchers turned adult human skin
cells directly into adult human blood
cells, without first returning them to a fully pluripotent state.
Fluorescence - activated sorting is then
used to identify and retrieve from this mix only those
cells that display strong reporter
gene expression, which represent the
cells with the most active enhancers.
«In addition, changes in how the
genes are expressed (turned on or off) could be
used in the future to predict how and when the cancer
cells will spread to other parts of the body and how fast they will grow.»
One of the crucial adaptations involved changes in a
gene that encodes a viral protein
used to enter
cells.
Researchers from Duke University had previously
used CRISPR to correct genetic mutations in cultured
cells from Duchenne patients, and other labs had corrected
genes in single -
cell embryos in a laboratory environment.
A virus containing the
gene that codes for two molecular «switches» essential for turning skin
cells into brown fat was
used to trigger the change (Nature, DOI: 10.1038 / nature08262).
«Many diseases, especially complex diseases, involve multiple
genes, and this system could be
used therapeutically to target and activate multiple
genes together and rescue these disease phenotypes,» says Albert Cheng, a graduate student in the Jaenisch lab and co-author of the
Cell Research paper.
By
using engineered zinc - finger nucleases (ZFNs) designed to target an integrated reporter and two endogenous rat
genes, Immunoglobulin M (IgM) and Rab38, we demonstrate that a single injection of DNA or messenger RNA encoding ZFNs into the one -
cell rat embryo leads to a high frequency of animals carrying 25 to 100 % disruption at the target locus.
For the first time, specialised
cells have been reprogrammed into the equivalent of embryonic stem
cells without
using genes that might trigger cancer.
«We are very excited about the prospect of
using these more physiologically relevant
cell - based models for
gene and small molecule drug discoveries,» says Dr. Andrew Liu, Assistant Professor of Biology at the University of Memphis.
DNA methylation is a mechanism
cells use to control
gene expression at the epigenetic level.
In 2006, he
used retroviruses to insert four
genes into the chromosomes of mouse skin
cells.
The survey, described today in a Policy Forum published by Science, randomly presented people with different vignettes that described genome editing being
used in germline or somatic
cells to either treat disease or enhance a human with, say, a
gene linked to higher IQ or eye color.
The scientists injected the USH1G
gene into the inner ear
using the innocuous AAV8 virus, which enabled them to specifically target the hair
cells.
The team
used viruses to replace the defective
genes that cause the anaemia with normal ones, then
used a second virus to insert
genes that «reset» the
cells to a pluripotent state.
Next, Anderson and his colleagues
used a set of genetic tools to identify exactly which neurons were responsible for the effect on aggression and to see if the
gene that encodes for Tk also controls aggressive behavior by acting in that
cell.
A team of researchers at the Stanford University School of Medicine has
used a
gene - editing tool known as CRISPR to repair the
gene that causes sickle
cell disease in human stem
cells, which they say is a key step toward developing a
gene therapy for the disorder.
Using the same technique, it should be possible to add other
genes to stem
cells to make them more efficient at different tasks.
Researchers then tested
cell cultures and mouse models by
using a
gene editing process called CRISPR - Cas9 to demonstrate how the presence or absence of myomaker and myomerger — both individually and in unison — affect
cell fusion and muscle formation.
These strands, known as oligonucleotides, take only a day or two to prepare and can be
used to create custom
genes to give
cells new functions.
The Porteus team started with human stem
cells from the blood of patients with sickle
cell disease, corrected the
gene mutation
using CRISPR and then concentrated the human stem
cells so that 90 percent carried the corrected sickle
cell gene.
CRISPR is normally
used to edit or delete
genes from living
cells.
Lee's team
used a retrovirus to transfer the fluorescent
gene to dog fibroblast
cells, but they could not control where the virus inserted the
gene.
The researchers
used the dead guide RNAs to turn on the Pdx
gene in the mice's livers, which caused the liver
cells to produce insulin, reversing the mice's diabetes.
«Controlling RNA in living
cells: Modular, programmable proteins can be
used to track or manipulate
gene expression.»
Vamsi Mootha, a mitochondrial biologist at Massachusetts General Hospital, his graduate student Isha Jain, and their colleagues
used a popular DNA - editing tool called CRISPR to knock out about 18,000 different
genes in human
cells that were altered to have the same problems as people with mitochondrial diseases.
The researchers say this finding may indicate that
cells use one strand to keep
genes intact and the other to evolve new, potentially advantageous features.
The so - called STEP trial, sponsored by pharmaceutical giant Merck & Co. and the federally funded HIV Vaccine Trials Network (HVTN), was the first to test the idea of stimulating the immune system's killer T
cells to hunt for the virus more aggressively, in this case
using a weakened form of the cold virus to carry three
genes from HIV.
Using a genome - wide genetic screen, Dr. Gelman and colleagues identified a previously unknown metastasis suppressor — the FOXO4 protein, which belongs to a family of
genes that are produced by all human
cells.
They will then reproduce as directed by their own
genes but
using the resources of the host
cell.
They then plan to
use a retrovirus to introduce the artificial
gene into tumour
cells.