Supplementary Materials

Supplementary Material for:

Liver-directed lentiviral gene therapy in a dog model of hemophilia B

Alessio Cantore, Marco Ranzani, Cynthia C. Bartholomae, Monica Volpin, Patrizia Della Valle, Francesca Sanvito, Lucia Sergi Sergi, Pierangela Gallina, Fabrizio Benedicenti, Dwight Bellinger, Robin Raymer, Elizabeth Merricks, Francesca Bellintani, Samia Martin, Claudio Doglioni, Armando D'Angelo, Thierry VandenDriessche, Marinee K. Chuah, Manfred Schmidt, Timothy Nichols, Eugenio Montini, Luigi Naldini*

*Corresponding author. E-mail: naldini.luigi@hsr.it

Published 4 March 2015, Sci. Transl. Med. 7, 277ra28 (2015)
DOI: 10.1126/scitranslmed.aaa1405

This PDF file includes:

  • Materials and Methods
  • Fig. S1. Lentiviral vectors efficiently transduce and regulate transgene expression in canine cells.
  • Fig. S2. Hematocrit, plasma fibrinogen, TAT, D-dimer, and clearance of lentiviral vector particles from blood after portal vein infusion in dogs with hemophilia B.
  • Fig. S3. Histology of liver biopsies from treated dogs with hemophilia B.
  • Fig. S4. Lentiviral vector content in liver biopsies and blood and sperm samples from treated dogs with hemophilia B.
  • Fig. S5. Factor IX expression and liver histology of tumor-prone mice or mice treated with a tumor promoter transduced with SIN.ET or ET.LTR lentiviral
    vectors.
  • Fig. S6. Comparison of the ET.LTR and SIN.ET CIS under different experimental conditions.
  • Table S1. Large-scale batches of lentiviral vectors.
  • Table S2. Blood cell counts and blood chemistry in M57.
  • Table S3. Blood cell counts and blood chemistry in O21.
  • Table S4. Blood cell counts and blood chemistry in O59.
  • Table S5. Lentiviral vector particles in swabs from dogs O21 and O59.
  • Table S6. Bleeding frequency in treated and untreated dogs with hemophilia B.
  • Table S7. Thromboelastography in treated dogs with hemophilia B.
  • Table S8. Inhibitor screen in treated dogs with hemophilia B.
  • Legends for tables S9 to S14
  • References (4751)

[Download PDF]

Other Supplementary Material for this manuscript includes the following:

  • Table S9 (Microsoft Excel format). Mice with hemophilia B transduced with SIN.ET as adults.
  • Table S10 (Microsoft Excel format). Integration sites retrieved from mice with hemophilia B transduced with SIN.ET as adults.
  • Table S11 (Microsoft Excel format). CIS identified in the data set of integration sites retrieved from mice with hemophilia B transduced as adults.
  • Table S12 (Microsoft Excel format). Cdkn2a−/−Ifnar1−/− and wild-type mice transduced with SIN.ET or ET.LTR as neonates.
  • Table S13 (Microsoft Excel format). Integration sites retrieved from tumor-prone mice transduced as neonates.
  • Table S14 (Microsoft Excel format). CIS identified in the data set of integration sites retrieved from tumor-prone mice transduced as neonates.

[Download Tables S9 to S14]