Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence

Spinal muscular atrophy (SMA) is characterized by progressive weakness of skeletal and respiratory muscles. This study aimed to evaluate the prevalence of pre-existing anti adeno-associated virus serotype 9 antibody (AAV9-Ab) titers among infantile-onset SMA diagnosed infants pre-screened for treatm...

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Veröffentlicht in:	Gene therapy 2023-02, Vol.30 (1-2), p.101-106
Hauptverfasser:	Aharoni, Sharon, Bistritzer, Jacob, Levine, Hagit, Sagi, Liora, Fattal-Valevski, Aviva, Ginzberg, Mira, Noyman, Iris, Cohen, Rony, Nevo, Yoram
Format:	Artikel
Sprache:	eng
Schlagworte:	13 13/1 42 42/44 631/378/1959 692/699/375 Age Antibodies Atrophy Biomedical and Life Sciences Biomedicine Cell Biology Child Copy number Demographics Demography Dependovirus - genetics FDA approval Gene Expression Gene Therapy Genetic engineering Genetic Therapy - methods Human Genetics Humans Immunization Infant Medical records Muscles Muscular Atrophy, Spinal - therapy Nanotechnology Neurology Neuromuscular diseases Patients Serogroup Skeletal muscle SMN protein Spinal Muscular Atrophies of Childhood - drug therapy Spinal Muscular Atrophies of Childhood - genetics Spinal muscular atrophy Viral infections Viruses
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