The discovery of the CF gene gave rise to the dream of a “cure” through gene therapy. Gene editing is one of the techniques being investigated; however, the method and its applications may not be understood within the affected portions of the general population; e.g., the CF community. To address that knowledge gap, the American Society of Gene & Cell Therapy issued a white paper entitled Therapeutic Gene Editing.
The introduction to the white paper notes:
Gene editing is a process that repairs or changes a gene. Medical researchers are exploring ways to use gene editing to treat or prevent human diseases. According to a recent public opinion survey, Americans are both excited and concerned about this. Given the complexity of gene editing and the rapid pace at which the science is progressing, it is no wonder that the public is both intrigued and perhaps uneasy.
The white paper is directed to the general public rather than medical professionals. Therefore, the document is a good basic resource for those who are curious about the science and regulation of gene editing.
You can read and/or download the white paper here: TherapeuticGeneEditingWP_Nov21_v1
A link to this white paper has also been added to our Information Resources, Medical Care page.