NF1 gene therapy

The history of gene therapy research for NF1 is unfortunately rather short.  I can find just one such instance, the work by Prof. David Segal of UC Davis to treat plexiform neurofibroma with undamaged NF1 genes delivered by a bacterial vector using transcription activator-like effector nucleases (TALE).  This one-year high-risk/high-reward effort was funded by the Army.   They concluded:

There are currently no medical treatments for plexiform neurofibromas. Several drugs are in clinical trial, but none affect the underlying genetic deficiency. This project represents the first therapeutic approach to restore normal levels of NF1 gene expression in the cells that support the tumor. ATFs can be considered a new paradigm in therapeutic development. ATFs work on a genetic level and can thus target “undruggable” proteins like NF1. We are also proposing to use tumor-infiltrating bacteria to continuously produce the ATFs at the tumor site until the tumor is gone. ATFs have never been delivered this way before. The ATFs are based on transcription activator like effector (TALE) transcription factors, which in nature are injected from bacteria into eukaryotic target cells. Therefore this project usurps the natural delivery system of these proteins to treat plexiform neurofibromas. Although we were not able to complete the testing objectives of this project, we were successful in developing the core enabling technology. This approach still represents a plausible and very different way to treat childhood neurofibromatosis, as well as other solid tumors.

In terms of final deliverables, they stated:

The PhD degree that Joshua Meckler anticipates to obtain next year will have been partially supported by this award. The bacterial TALE-ATF delivery system developed from this award will likely find future use by us and other investigators.

Prof. Segal and his student also published a paper on Genome Engineering at the Dawn of the Golden Age.  According to his LinkedIn, Mr. Meckler is still working on his thesis.

 

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