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The investigational adeno-associated virus (AAV) genetherapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.
The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL GeneTherapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL GeneTherapy Platform. Related: Red Meat Allergy Test Gets FDA Clearance. “We
These modifications regulate geneexpression without changing the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. It’s a great tool for controlling geneexpression.”.
Further, the transcription factors contain two molecular domains, the DNA binding domain and the activation domain. Moreover, some transcription factors contain additional domains, such as ligand binding domain in order to interact with chemical signals.
These modifications regulate geneexpression without altering the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. It’s a great tool for controlling geneexpression.”.
I was recently reminded of my journey out of the HCT nightmare as I watched a documentary about genetherapy. Genetherapy is a huge deal – some genetherapies are already achieving jaw-dropping results for people with previously untreatable illnesses. It’s fascinating stuff. So long, HCT.
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