Gene Transfer, Targeting
and Therapeutics Core

Recent Papers of Interest

• Selective viral vector transduction of ErbB4 expressing cortical interneurons in vivo with a viral receptor–ligand bridge protein., Choi et al., Proc Natl Acad Sci
• Monosynaptic Rabies Virus Reveals Premotor Network Organization and Synaptic Specificity of Cholinergic Partition Cells. Stepiene et al., Neuron
• The next step in gene delivery: Molecular engineering of adeno-associated virus serotypes. Faust et al., J Mol Cell Cardio
• Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction. Finn et al., Mol Ther
• The neonatal blood-brain barrier is functionally effective, and immaturity does not explain differential targeting of AAV9. Saunders et al., Nat Biotech
• Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Duque et al., Mol Ther
• High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Petrs-Silva et al., Mol Ther
• Comparative analysis of HIV-1-based lentiviral vectors bearing lyssavirus glycoproteins for neuronal gene transfer. Federici et al., Genet Vaccines Ther

Useful Links

• Production and purification of lentiviral vectors – Verma Lab
• Production and characterization of recombinant adeno-associated viral vectors – Samulski lab
• MTA for Samulski rAAV vectors – UNC gene therapy core
• Contact information for U. PENN vector core – MTA for AAV serotypes 8,9 and rh10.
• Lenti-web – information and discussion groups about lentiviral vectors
• Addgene – distribution of useful plasmids including 2nd and 3rd generation lentiviral packaging systems.
• Open Biosystems – Human and mouse whole genome lentiviral shRNAmir vectors – pGIPZ and the tet-inducible pTRIPZ.