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Human Gene Therapy Methods|June 18, 2013
RD2-MolPack-Chim3, a packaging cell line for stable production of lentiviral vectors for anti-HIV gene therapyAnna Stornaiuolo, Bianca Maria Piovani, Sergio Bossi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 21, 2007
Long-term engraftment of single genetically modified human epidermal holoclones enables safety pre-assessment of cutaneous gene therapyFernando Larcher, Elena Dellambra, Laura Rico, et al.
Molecular Therapy. Nucleic Acids|June 19, 2017
Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 SystemAnnalisa Lattanzi, Stephanie Duguez, Arianna Moiani, et al.
International Journal of Molecular Sciences|July 27, 2022
Engineered Sleeping Beauty Transposon as Efficient System to Optimize Chimp Adenoviral ProductionSamantha Baldassarri, Daniela Benati, Federica D'Alessio, et al.
Haematologica|March 8, 2005
Stem cell plasticity: time for a reappraisal?Roberto M Lemoli, Francesco Bertolini, Ranieri Cancedda, et al.
Neurobiology of Disease|March 4, 2008
Generation of a alpha-synuclein-based rat model of Parkinson's diseaseAlessandra Recchia, David Rota, Patrizia Debetto, et al.
Molecular Therapy. Methods & Clinical Development|May 1, 2018
Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined ImmunodeficiencyValentina Poletti, Sabine Charrier, Guillaume Corre, et al.
Stem Cell Reports|April 7, 2016
Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte DifferentiationAlessia Cavazza, Annarita Miccio, Oriana Romano, et al.
Molecular Therapy. Methods & Clinical Development|October 29, 2015
Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophySilvia Ungari, Annita Montepeloso, Francesco Morena, et al.
Molecular Therapy. Methods & Clinical Development|December 12, 2018
Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell DiseaseValentina Poletti, Fabrizia Urbinati, Sabine Charrier, et al.
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