Final Program - American Society of Gene & Cell Therapy

Program Schedule, Wednesday, May 15, 2013
Wednesday, May 15, 2013
Education Session 124
1:15 pm - 2:45 pm
ROOM: 150 ABC
Topical Review: Synthetic Gene Delivery
An overview of different philosophies of synthetic delivery systems for nucleic acids. The irst talk will address silk elastin-like proteins for
gene delivery and how they can be combined with virus for an enhanced hybrid gene delivery vehicle. The second talk will focus on “smart”
pH-sensitive polymeric carriers for therapeutic delivery of nucleic acids. The inal talk will examine cell-penetrating peptides for retinal gene
delivery.
CHAIR: James Yockman, PhD
SPEAKERS
Hamid Ghandehari, PhD
Recombinant Polymers for Gene Delivery
Polymeric systems have shown utility in gene delivery. They can form complexes with nucleic acids or be used as matrices for
controlled release. Majority of polymers used for such purposes are synthesized by chemical strategies that result in limited control
over polymer structure and therefore function. Recombinant polymers provide exquisite control over backbone structure. This talk
will summarize the use of recombinant polymers for gene delivery applications.
You Han Bae, PhD
pH-Sensitive Polymeric Carrier for Nucleic Acid Delivery
The journey of a gene carrier from an administration site to target individual cells encounters a series of formidable barriers in
the blood compartment, when crossing vascular walls, and while penetrating extracellular matrix. Cell uptake and intracellular
traficking steps then follow, which have been major foci in carrier development and in vitro tests. The carrier may require
switching surface property depending on environmental conditions during the voyage for higher probability of reaching its target
cell and action site. The potential roles of pH-responsive polymers, particularly weakly anionic polysulfonamide and cationic
poly(L-histidine), in this challenging journey will be discussed.
Rajendra Kumar-Singh, PhD
Pegylated Peptide-DNA Nanoparticles Delay the Onset of Retinal Degeneration in Mouse Models of Retinitis
Pigmentosa
We have developed a novel peptide referred to as ‘Peptide for Ocular Delivery’ or POD that can penetrate cells and tissues in vivo
within 5 minutes. When pegylated, POD can compact DNA and enable marker or therapeutic gene delivery to retina in vivo. These
nanoparticles are approximately 150 nm in size and can enable therapeutic transgene expression in vivo for more than 70 days.
POD-DNA nanoparticles can reduce apoptosis and delay retinal degeneration in a mouse model of retinitis pigmentosa. POD has
potential therapeutic applications in ocular as well as non ocular organ systems.
Education Session 125
1:15 pm - 2:45 pm
ROOM: 151 ABCG
Topical Review: The Ins and Outs of Integration Deicient Lentiviral Vectors (IDLV’s)
The Ins and Outs of Integration Deicient Lentiviral Vectors (IDLV’s)”, will focus on the development and utilization of IDLV’s in preclinical
trials. The session comprises three presentations. The irst is an introductory presentation, which will concentrate on the molecular
mechanisms involved in the production and regulation of gene expression from IDLV’s. The second presentation will focus on illegitimate
integration of IDLV’s, and the third presentation will describe the development of a novel IDLV system for cancer immunotherapy.
CHAIR: Tal Kafri, MD, PhD
SPEAKERS
Tal Kafri, MD, PhD
The Basics of Integration Defective Lentiviral Vectors
This introductory presentation will focus on the molecular mechanism involved in the generation of integration defective lentiviral
vectors (IDLV’s) and the molecular assays employed to determine the eficacy and safety of the currently used IDLV gene delivery
systems. In addition the presentation will outline advancement in IDLV design, which improve IDLV gene expression and reduce
illegitimate integration.
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Final Program SALT LAKE CITY, UTAH May 15–18, 2013