To use gene therapy to replace retGC1 to restore visual function

Not so long ago there was a research about using gene therapy to replace retGC1 to restore visual function. In the research with recombinant proteins, mice lacking the protein retGC1, which is deficient in humans suffering Leber congenital amaurosis-1 (LCA1), a disorder that causes severe visual impairment beginning in infancy, received gene therapy to replace retGC1 and showed fully restored visual function that persisted for at least 6 months. The success would strongly support clinical testing of a gene therapy targeted to the retinas of LCA1 patients as the researchers concluded.

Sanford Boye, Shannon Boye, and coauthors from University of Florida College of Medicine, Gainesville, University of Oklahoma College of Medicine, Oklahoma City, and Salus University, Elkins Park, PA, are the authors of the article. They emphasize the need for a treatment strategy targeting the loss of cone function that occurs in the eyes of patients with LCA1. They describe a gene replacement approach that uses an adeno-associated viral (AAV) vector to deliver the gene encoding the retGC1 protein to the cone-rich central retina in an all-cone mouse model deficient in retGC1. They report their results conclusions in the article.

"This study shows the tremendous potential of recombinant (rAAV) gene therapy for the effective treatment of genetic causes of vision loss," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA.

This article about the Gene therapy is available free on the Human Gene Therapy website before September 30, 2015. Want to know more? Go and see! Flarebio Biotech LLC is a National High-Tech Enterprise with research, production and sales as one. It offers recombinant proteins like recombinant KEL at good prices.

What is it that leads cells to differentiate into T cells?

Every cell in our body has its own fate. They may become skin cells or perhaps other types of cells, and all these are determined by genes. Scientists from California Institute of Technology have further studied the differentiation of T cells. T-lymphocytes are cells secreted by thymus can swallow the virus-infected cells. Similar to using T cell transformation to promote recombinant proteins such as recombinant horse proteins and recombinant dog proteins, the researchers hope to be able to figure out what leads cells to differentiate into T cells.

The study was published in Nature Immunology on July 4th. The lead author and postdoctoral at the school Hao Yuan Kueh said, "We have already known which kind of genes that control cells' differentiation into T cells, but we want to know what original factors are that make this gene play a role."

They found a group of DNA-binding proteins which contains four proteins and they controlled the opening and closing of T cell genes by overlapping layers. This finding is really a great breakthrough, because in the past, our conception of the role of transcription factors is instantaneous. The study could help doctors get a lot of T cells, which has great application significance to viral infectious diseases including AIDS.

Laboratory staff Kueh said, "In the past, we know that the regulation of genes needs the involvement of a variety of transcription factors, and so it is also the same with T cell differentiation. Multifarious transcription factors can ensure that the right expression of cells."

The authors pointed out that the key of this finding is the real-time video of living cells. They used genetically-engineered mice to conduct fluorescence detection on proteins. The transgenic mice were introduced with Bcl11b genes. When this kind of genes is activated, tissues of mice would shine. Through detection of different transcription factors and proteins, the researchers can distinguish the role of different proteins.

These four proteins together play a role in the process of opening the genes, of which the first two proteins (TCF1 and GATA3) opened the door, Notch protein switched the switch and the fourth protein Runx1 amplified the signal. Flarebio is a biotech company which is specialized in research, production and sales of recombinant proteins (including recombinant KEL) and antibodies and other related products.

The folding mechanism is a part of the genetic code

Theoretical physicists have recently identified biological sensational news: our body's genetic information is not only stored in the nucleotide sequence of the DNA and it partly depends on the way DNA folds into chromosomes. Specifically, the three-dimensional structure of DNA will determine gene expression. In fact, the biologists are already aware of the things above and with the use of recombinant proteins, and they even have found the proteins which assist to fold DNA. But now it is a bunch of physicists who for the first time reveal the true mystery and what they use are computer simulation tools.

Watson and Crick discovered the double helix structure of DNA in 1953 and determined our genetic information is stored in the base pairs composed of four basic groups - G, A, C, T. These sequences will determine which kind of protein will be produced in which cell. Some DNA in all the cells in our body is the same at first, but why they can differentiate different proteins to meet so many different organ functional needs? Our stomach cells may not produce proteins that can make the eyes brown, but they need to produce digestive enzymes. Then how does such a division mechanism work? It is a complex question which needs a lot of experiments using the technology of recombinant protein to solve out.

In the 80's, scientists found that the mechanism was controlled by the folding way of DNA inside the cells. Besides, environmental factors are also significant. For example, environmental stress can activate and silence some genes. DNA folding way determines which genes can be "read" in the cells. To make it simple, the genes folded in the most remote corner are "unreadable" and genes on the outside can be "read". In this way, different cells can determine which protein that should be synthesized to meet different needs. Prior studies have shown that the mutation ways of DNA are more than one: in addition to changing the DNA nucleotide sequence, changing DNA folding is another way. Changing the shape and the folding of DNA bases and the "read" nature, the final protein synthesis will also be affected. Recombinant KEL can show the process.

More and more scientists are doing research in this area. With the understanding of controllable DNA folding, the possibility that we can finally take advantage of it is getting a little bit larger. Related articles have been published in the academic journal PLOS ONE.