T Cell Gene Therapy : The Center Of Research With Stem Cell Transfection In Geney Cells

For many years biomedical researchers like myself have been trying to create more proactive ways to amend the genome for living cells. In more recent fieldwork studies there has been a new state of the art instrument based on bacterial CRISP in close works with protein 9 often referred to as CAS9 from the streptococcus progenies have possibly unlocked new data. The CRISP/CAS9 tries to manipulate the function of the gene using homologous recombination and RNA interference, but is set back because it can only provide short term restriction of the genes function and it’s iffy off- target effects.

CRISPR is a new gene-modifying tool that has the potential to treat numerous medical conditions by editing genes that are responsible for certain diseases. This technology is based on the ability of bacteria to destroy the DNA of invading viruses. Studies have suggested that this new technology can be applied to human cells, although the idea of chopping up regions of the human genome can be unethical and could even be harmful. In order for the treatment to be administered to a patient, a small piece of RNA and an enzyme that makes a cut in the DNA are delivered to the cells. A biotechnology company, known as Editas Medicine, located in Cambridge, MA, is already designing treatments for conditions of the blood and the eye using CRISPR. For

More recently, Kang et al have employed a different approach using a non-viral delivery method for CRISPR-Cas, known as the

From the science community perspective, the CRISPR-Cas system could reduce or even eliminate many of the difficulties researchers face when gene editing such as cost, duration and accuracy. Prior to CRISPR-Cas, gene editing was performed in “big labs” with experts

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeat, referring to the repeating DNA sequences found in the genomes of microorganisms. CRISPR technology allows scientists to make precise changes in genes by splicing and replacing these DNA sequences with new ones. Through these changes, the biology of the cell is altered and possibly affects the health of an organism. The possibilities are endless as this offers opportunities in curing deadly diseases, modifying genes, and changing humanity as we know it. Although bioengineering has been around since the 1960s, CRISPR is significant because of the comparative low costs and the ease of the procedure to

The CRISPR Team was fortunate to be a part of the “Virus Documentary” (SciChannel) and conduct successful experiments discovering the activity of viruses. Through a series of test conducted by The CRISPR Team, it

The ability to engineer biological systems and organisms has an enormous potential for applications across basic science, medicine and biotechnology. Genome editing is a group of technologies that allow scientists the ability to change an organism’s DNA, which can provide better outcomes for health and disease control compared to natural immunity and mutations. Genome editing (gene editing) allows genetic material to be added, removed or altered at particular locations in the genome. A number of gene editing technologies have emerged in recent years with one of the most versatile and precise methods of genetic manipulation being Crispr-Cas9 (Steve scott,2016). The term Crispr-cas9, (clustered regular interspaced short palindromic repeats)

Surrounded by patent and ethical issues lies a gene editing method with massive potential within the biotechnology industry. The CRISPR-Cas9 system works like ‘molecular scissors’ where Cas9 is an endonuclease that targets a specific DNA sequence (Griggs. 2015). This is more efficient than the previous methods such as zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), as well as being simpler to use. CRISPR-Cas9 uses single guided RNA (sgRNA) to reach the desired gene, where it is able to cleave the double stranded DNA in the presence of a Protospacer Adjacent Motif sequence (PAM sequence) (Ran et al. 2013). This is the stage where the gene can be altered via the cells own repair system due to the break in the DNA (Nehme et al. 2014). Solutions to sickle-cell anaemia, malaria and beta thalassemia are just a few of the life changing impacts this method could have in the future.

The CRISPR/Cas9 system is an accurate, cheap, and fast way to edit genes. It is used to edit parts of the genome by removing, adding, or altering some sections of the DNA sequence. The CRISPR/Cas9 system was used with HIV cell lines and RNA-directed gene editing to prevent new HIV-1 infections. Both experiments resulted in the stopping of the reactivation of the HIV infection.

In recent studies, scientists have taken mice who have muscular dystrophy and have injected them with theses CRISPR-Cas9 viruses. After these same mice were put through a test for strength and performed better than mic who were not treated by these

magine, 20 years from now, sitting in a cold doctor's office deciding the genes of your unborn baby, what color hair, eyes, speed of metabolism, height would you even know what to pick? Impossible you might say but in this day and age technology is growing ever so rapidly that picking the genetic makeup of your baby is closer than you might think. The technology is called CRISPR. This technology doesn't only have the ability to change physical traits, but genetic traits specifically genetic abnormalities and diseases. 20 years ago, no one would have ever thought we would have the answer to, in theory, cure every genetic disease from sickle cell anemia to cystic fibrosis. However, with great scientific breakthroughs comes questioning and

Hsu, P., Lander, E., & Zhang, F. (2014). Development and Applications of CRISPR-Cas9 for Genome Engineering. Cell, 157(6), 1262-1278. http://dx.doi.org/10.1016/j.cell.2014.05.010

There are three techniques that researchers are working on. The first and most common is ex vivo ( or "outside the living body") therapy. The defective cells are removed from the patient and replaced with the normal DNA before returning to the body. This therapy targets the blood cells because many genetic defects alter the functioning of one type of these cells or another. But since blood cells have limited life spans follow-up treatments are required. Future efforts will most likely target stem cells of the bone marrow. Stem cells are ideal for gene therapy because they appear to be immortal. Researchers have obtained stem cells from human bone marrow, but they are having difficulties getting genes into the cell as well as inducing the cells to produce many new blood cells (Anderson, 1995).

Gene editing with CRISPR-Cas9 will be the next cure for cancer, many other diseases and could change many lives. Even though this ground breaking technology has not been put to use on humans yet, when it does it will be well worth the wait. Many countries have been working on the CRISPR-Cas9 for months to allow it to be used to cure the many diseases that can not be cured with modern technology.

Once the complex was bound to the DNA, a cut would be made to eliminate and destroy the invaders. 83% of archaeal genomes and 45% of bacterial genomes (Shabbir, M. et al, 2016) were shown to be able to successfully utilize the CRISPR Cas9 system. These are very promising statistics, so it is no wonder that there has been such an advancement in the past few years to bring this technology to eukaryotic cells, mammalian cells and eventually human cells.