Gene Therapy

Nascent Transcript Author: Anna Miller A recent decision by the pharmaceutical company Novartis to provide 100 free gene therapy treatments for spinal muscular atrophy (SMA), a debilitating yet rare genetic condition, to patients selected through a lottery program has sparked debate about the ethical implications. SMA is a rare autosomal recessive genetic disorder caused by... Read More

In 2000, recognizing the involvement of ASHG members in gene therapy research and implementation, ASHG issued a statement commenting on the need for rigorous research and consideration of patient interests. Related: Statement on Gene Therapy (August 2000) Read More