Navigation Links
New Platform for Regulating Expression of Therapeutic Genes Unveiled

A new platform for regulating the expression of therapeutic genes, which will have major implications for gene and cell therapy, was presented today at the 10th Annual Meeting of the American Society of Gene Therapy (ASGT) in Seattle.

Carefully regulating gene expression is crucial to the success of gene and cell therapy. For many applications, gene transfer is being employed to engineer cells for therapeutic factor production, expansion, selection of a desired cell type and even fail-safe suicide. These applications often require precise regulation in order to ensure gene expression in the correct tissue and prevent it in unwanted cell types.

Now, a team of scientists led by Dr. Luigi Naldini at the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) in Milan have developed a new platform. Dr. Naldini and colleagues provided a striking demonstration that their new design can enable delivered genes to become highly responsive to a cells identity. This is particularly relevant for the emerging field of stem cell gene therapy, in which genes are delivered into a cell that can give rise to many distinct cell types.

To achieve their goal, the TIGET scientists came up with a strategy to take advantage of a recently discovered network of gene regulation mediated by small RNA molecules, known as microRNA.

MicroRNAs downregulate the expression of specific genes in cells where the gene is not needed, and thereby have an important influence over the identity of the cell. More than 350 mammalian microRNAs have been identified, with many being present only in some specific tissues and cell types.

Dr. Naldinis group showed that the addition of microRNA binding sites into their gene delivery vectors results in gene regulation dictated by the cells own cognate microRNA. Simply put, they could engineer their gene to be turned off in cells where the microRNA is present. As a proof-of-principle, the resea rchers used a lentiviral vector to introduce a gene encoding a fluorescent protein into human embryonic stem cells. Their gene contained target sites for a microRNA specific of neuronal cells. While undifferentiated, the embryonic stem cells expressed high levels of the fluorescent gene, however, as soon as the cells turned into neurons, the engineered gene was turned off in response to the presence of the microRNA in the neurons. So versatile is their approach, that they could even demonstrate the reverse. By changing the microRNA binding site in their vector to one recognized by an embryonic stem cell-specific microRNA, their vector was kept off in immature embryonic stem cells, but began to express itself as the cell turned into more defined tissues. This new platform now provides a means for scientists to achieve highly tailored gene expression patterns when delivering recombinant genetic material into cells.

We had been looking for ways to regulate transgenes better, when it hit us, nature had already solved the problem for us, says Dr. Brian Brown, one of the studies authors. Now, we can utilize a cells own regulatory network to control a gene we introduce.

Dr. Naldinis group has already begun to successfully exploit microRNA regulation for achieving stable long-term correction of hemophilia in the mouse model and for improving the safety of hematopoietic stem cell gene therapy.

The American Society of Gene Therapy is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of gene and related cell and nucleic acid therapies and the promotion of professional and public education in the field.


'"/>




Related medicine news :

1. Lovers Latest Hang Outs – Railway Platform
2. Substance P May Pre-empt VIP in Regulating Temperature
3. Controlled Gene Expressions Can Cure Many Diseases
4. Genetic Expressions Can Influence The Process Of Aging
5. Non-Coding RNA Vital For Gene Activation And Protein Expression
6. Protein Expression to Serve as Marker for Head and Neck Cancer
7. Innocent Expression of Compassion, Children Draw For HIV kids
8. New Diagnostic Tool Uncovered Gene Expression Patterns in Recessive Genetic Disorders
9. Facial Expressions are Inherited: Study
10. Telomerase Expression Linked To Body Mass
11. How Blood Flow Dictates Gene Expression
Post Your Comments:
*Name:
*Comment:
*Email:


(Date:1/24/2017)... ... January 24, 2017 , ... ... to Thrive , Well-meaning studies such as the Fordham Institute’s High Stakes ... serve top students, such as including gifted or high-achieving students as a subgroup ...
(Date:1/24/2017)... (PRWEB) , ... January 24, 2017 , ... ... Pollack, Ph.D., http://www.faculty.washington.edu/ghp , Sharon Kleyne, the nation’s foremost water advocate ... Global Climate Change and Your Health on Voice America, once again welcomed one ...
(Date:1/24/2017)... ... ... A yellow fever outbreak in Brazil has infected over 100 people and claimed ... contact with infected mosquitos. The outbreak has sparked increased concern about the virus and ... multiple health organizations, the best way to prevent yellow fever is through vaccination. For ...
(Date:1/24/2017)... ... January 24, 2017 , ... ... topics like finances, friendship, marriage, leadership, gossip, prostitution, adultery, anger, and common sense. ... authors, Dr. Judith Coats and Dr. David Coats. In September of 1983, they ...
(Date:1/24/2017)... ... , ... “Mysteries Revealed On Speaking In Tongues”: an engaging and dynamic ... Christians. “Mysteries Revealed On Speaking In Tongues” is the creation of published author, Tina ... located in Michigan. , “We need to partner with Jesus and be the ...
Breaking Medicine News(10 mins):
(Date:1/24/2017)... India , Jan. 24, 2017 Market Research Future has ... Market for Wound Closure Device is growing rapidly and expected to continue ... ... at a CAGR of 5% from 2013 to 2019 and reaching a ... of the forecasted period, 2016-2022 Global Wound Closure Device Market ...
(Date:1/24/2017)... Nev. , Jan. 24, 2017  The ... that specializes in high-value orthopaedic implants, announced the ... today. The OIC Tibial Nail ... tibia. Strategically placed proximal and distal screw holes ... hole that allows dynamization.  The nail is available ...
(Date:1/24/2017)... 2017 Trifecta Clinical , a leading ... Rick Ward to Vice President of Commercial ... also announcing the promotion of Ericka Atkinson ... Rick joins Trifecta from Greenphire where he was ... business development positions within the healthcare industry throughout his ...
Breaking Medicine Technology: