Paola Luzi

A large deletion together with a point mutation in the GALC gene is a common mutant allele in patients with infantile Krabbe disease

Mohammad A. Rafi, Paola Luzi, Yue Qun Chen et al. · Human Molecular Genetics · 1995 · 119 citations

Life Sciences, Genetics, Molecular Genetics +10

Extended Normal Life After AAVrh10-mediated Gene Therapy in the Mouse Model of Krabbe Disease

Mohammad A. Rafi, Han Zhi Rao, Paola Luzi et al. · Molecular Therapy · 2012 · 114 citations · Full text

Medicine, Neurology, Neuroscience +14

AAV-Mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy

Mohammad A. Rafi, Han Zhi Rao, Marco A. Passini et al. · Molecular Therapy · 2005 · 110 citations · Full text

Life Sciences, Neurobiology, Neuroscience +17

Structure and organization of the human galactocerebrosidase (GALC) gene

Paola Luzi, Mohammad A. Rafi, David A. Wenger · Genomics · 1995 · 91 citations

Life Sciences, Biochemistry, Molecular Biology +12

Characterization of the large deletion in the GALC gene found in patients with Krabbe disease

Paola Luzi, Mohammad A. Rafi, David A. Wenger · Human Molecular Genetics · 1995 · 84 citations

Life Sciences, Genetics, Molecular Genetics +10