Sentences with phrase «effective gene therapy approaches»

With the aim of pioneering a safe, effective gene therapy approach, St. Jude scientists have spent years developing an innovative vector for X-linked SCID.

Not exact matches

As the central nervous system is also a target of AAV vectors, Hélène Puccio and Patrick Aubourg's teams are investigating whether a similar approach using gene therapy could be as effective for the spinal cord and cerebellum as it is for the heart.

«Because this mutation already exists in nature and is benign, this «organic gene therapy» approach should be effective and safe to use to treat, and possibly cure, serious blood disorders.

This approach for gene therapy to treat fatty liver disease, for example, might prove both safer and more effective than reengineering cells in the liver itself.

While effective, the downside to these approaches to vision rescue is that each disease requires its own form of gene therapy to correct the particular genetic mutation involved, a time consuming and complex process.