부산대학교 도서관

For later-onset phenotypes of SMA, "you might have a patient who could feasibly make a decision for himself or herself" about treatment, Dr. Bateman-House said, while treating preemptively in infancy might result in the patient forfeiting their autonomy. Moreover, gene therapy is delivered in viral vectors to which an immune response will develop: A person who has 4w received a treatment using one viral vector cannot likely receive sn d seaeiu A @9 >ioo S BueAu A 0 either a repeat dose or a potentially better therapy using the sarne vector. RARE NEUROLOGICAL DISEASE SPECIAL REPORT Lin-l wwollowl Ethics meets practice in gene therapy for neuromuscular disease Four experts- Alison Bateman-House, PhD; Lesha D. Shah, MD; Jennifer Kwon, MD; and Richard Finkel, MD- discuss how ethical concerns play out in the clinic over the use of gene therapy and antisense treatments in young children and infants. [Extracted from the article]