Author

J. Baruteau

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Open access Aug 2026

CCDC149: a novel gene associated with hypopituitarism and neurodevelopmental impairment.

This study is the first to report CCDC149 variants in association with Congenital Hypopituitarism, supporting the possibility of impaired ciliary function as an underlying mechanism in this complex disorder.

L. Gregory, Shoshana Rath, Hanna Mandel et al. · European Journal of Endocrin... · 0 citations

Open access Jul 2026

Identification of AAV Vector Integration Sites in Primary Human and Macaque Hepatocytes in vivo, and Analysis of Oncogenic Risk.

RAAV integration patterns in primary human hepatocytes xenografted into FRG mouse livers and in hepatocytes from cynomolgus macaques following systemic rAAV administration support a low oncogenic risk profile for the evaluated vector while reinforcing the value of direct human liver integration analyses to refine risk assessment and guide the development of safer gene therapy platforms.

S. Scott, C. Hallwirth, Natsuki Sasaki et al. · Molecular Therapy · 0 citations