Sentences with phrase «therapeutic genes into»

Taking advantage of this, researchers can insert therapeutic genes into a virus, then use the viruses to shuttle the genes to the appropriate cells or tissues inside a human body.

Finding a safe way to get therapeutic genes into the cells has been a major challenge, however.

The virus inserts the therapeutic gene into the cell's DNA and uses its instructions to produce a receptor protein — a modified version of a common glutamate receptor ion channel - that they display on their surface.

Not exact matches

This fact is rooted in a packaging problem: the gene encoding FIX is much smaller than the gene for FVIII, and better fits into a vector, usually a harmless virus, designed to deliver therapeutic genes to a patient.

For gene therapies, scientists remove most of the AAV genome, replace it with therapeutic genetic cargo, and inject trillions of copies into the patient.

The vector then unloads its genetic material containing the therapeutic human gene into the target cell.

Like mRNA, gene therapy can induce cells to make therapeutic proteins, but it typically introduces DNA that can integrate unpredictably into the genome.

Crucially, only the therapeutic gene is incorporated into the hybrid virus, so it can not replicate.

In the study published in the journal Science Signaling, the team led by LLuís Espinosa, investigator of IMIM's research group into stem cells and cancer, have shown that inhibition of endosomal activity is a potential therapeutic strategy for the treatment of cancers with the BRAF mutated gene.

Carrying the therapeutic genes, AdV made it into the patients» livers.

Here, the researchers delivered a therapeutic gene along with a short hairpin RNA (shRNA) into liver cells in mice.

Their work could overcome a stumbling block for gene therapy by ensuring that a gene is actually inserted into a target cell's chromosome, where it will order the production of therapeutic proteins.

Gene therapy offers the possibility of replacing the function of the defective or missing gene with that of a therapeutic gene inserted into the cGene therapy offers the possibility of replacing the function of the defective or missing gene with that of a therapeutic gene inserted into the cgene with that of a therapeutic gene inserted into the cgene inserted into the cell.

Since then he has continued to investigate new strategies to overcome the major hurdles to safe and effective gene transfer, translate then into new therapeutic strategies for genetic disease and cancer, and allowed new insights into hematopoietic stem cell function, induction of immunological tolerance and tumor angiogenesis.

The discovery of new genes involved in these repair mechanisms is very important in order to understand the development of these tumours and delve more deeply into their therapeutic vulnerabilities.

A DNA - covered submicroscopic bead used to deliver genes or drugs directly into cells to treat disease appears to have therapeutic...

The condition is more common than muscular dystrophy and cystic fibrosis, but the development of new therapeutic concepts is hindered by the fact that unlike muscular dystrophy and cystic fibrosis, where a single mutated gene causing the disease is known, the entire human chromosome 21 (containing around 300 genes) still has to be dissected into individual gene - dose contributions to the DS symptoms.

SOX2 is one of two key genes researchers use to generate induced pluripotent stem cells (iPSCs), which are capable of differentiating into all cell types for research and potential therapeutic applications.

Fundamental Science Overview Gene and gene - modified cell therapies treat diseases by delivering therapeutic DNA into a patient's cells, either in (in vivo) or outside his or her body (ex viGene and gene - modified cell therapies treat diseases by delivering therapeutic DNA into a patient's cells, either in (in vivo) or outside his or her body (ex vigene - modified cell therapies treat diseases by delivering therapeutic DNA into a patient's cells, either in (in vivo) or outside his or her body (ex vivo).

The first step in both direct and cell - based gene therapy is packaging a therapeutic transgene into a delivery vehicle (e.g., a viral vector), followed by expansion of its host cell lines to produce high - enough vector concentrations.

This overview describes how newly identified genes implicated in human neurodegenerative diseases are making their way into the easy - to - manipulate fruit fly and providing new insights into disease pathology and therapeutic avenues.

Gene therapy a therapeutic approach in which engineered genes are introduced into a patient's cells to treat a disease.