Fanconi anemia and βc deficiency-associated pulmonary alveolar proteinosis as two hereditary diseases of childhood which are potentially curable by stem cell gene therapy but require different therapeutic approaches

Uta Dirksen, T. Moritz, S. Burdach, M. Flasshove, H. Hanenberg

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4 Scopus citations

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Medicine and Dentistry

Pharmacology, Toxicology and Pharmaceutical Science

Biochemistry, Genetics and Molecular Biology