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References
2.
Kim
J
,
Hu
C
,
Moufawad El Achkar
C
, et al. Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease . New Engl J Med , 2019 ; 381 :1644 -1652 .
3.
Synofzik
M
,
van Roon-Mom
W
,
Marckmann
G
, et al. Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives . Nucleic Acid Therapeutics , 2022 ; 32 : 83 –94
