S. Penna

Autosomal recessive osteopetrosis: mechanisms and treatments

Valentina Capo, A. Villa, S. Penna, 2021, Disease models & mechanisms.

One Disease, Many Genes: Implications for the Treatment of Osteopetroses

Valentina Capo, A. Villa, E. Palagano, 2019, Front. Endocrinol..

Two mutations in mitochondrial ATP6 gene of ATP synthase, related to human cancer, affect ROS, calcium homeostasis and mitochondrial permeability transition in yeast.

R. Kucharczyk, D. Płochocka, Małgorzata Lichocka, 2018, Biochimica et biophysica acta. Molecular cell research.

Efficacy of lentivirus‐mediated gene therapy in an Omenn syndrome recombination‐activating gene 2 mouse model is not hindered by inflammation and immune dysregulation

L. Notarangelo, K. Dobbs, G. Wagemaker, 2017, The Journal of allergy and clinical immunology.

Expanded circulating hematopoietic stem/ progenitor cells as novel cell source for the treatment of TCIRG1 osteopetrosis

A. Schulz, C. Carlo-Stella, R. Cusano, 2020, Haematologica.

Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations

Valentina Capo, M. C. Castiello, P. Uva, 2023, Frontiers in immunology.