The successful treatment of selected deleterious genetic diseases using gene therapies

01 Sep 2022
The successful treatment of
selected deleterious genetic
diseases using gene therapies
Patients with rare genetic diseases usually have limited treatment options if any is available. Gene therapy has been progressing tremendously especially in the treatment of challenging rare genetic diseases such as spinal muscularatrophy (SMA). Modification of genetic diseases perhaps to the point of cure may be possible with gene therapy. Two renowned experts in clinical genetics discuss gene therapies for selected deleterious genetic diseases
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