Final Program - American Society of Gene & Cell Therapy

Program Schedule, Thursday, May 16, 2013
Nicholas Restifo, MD
Curative Cancer Immunotherapy Using Stem cell-like T cells
T lymphocytes play a critical role in the immune response against infectious disease and cancer. The recently identiied T memory
stem cells (TSCM) are endowed with the stem cell–like attributes of self-renewal capacity and multipotency. These qualities make
TSCM an attractive subset to employ in adoptive immunotherapies for cancer because they might overcome current limitations,
including ineficient T-cell engraftment, poor persistence, and inability to mediate a prolonged immune attack. TSCM display robust
proliferative and survival capacities and were superior to other memory T-cell subsets in causing regression of large, established
tumors in preclinical models. Protocols to facilitate translation of TSCM into the clinic will be discussed.
Jeffrey S. Miller, MD
Educating Natural Killer Cells to Kill Cancer
Natural killer (NK) cells express a number of activating and inhibitory receptors to determine their function through a process
called NK cell education. We have shown that adoptive transfer of haploidentical NK cells can induce remissions in some
patients with refractory acute myeloid leukemia (AML). Limitations of this therapy include host immune rejection, how to
maximize the persistence and expansion of donor NK cells and overcoming regulatory T-cell suppression. Lastly, donor Killer-cell
Immunoglobulin-like Receptor (KIR) genes may inluence NK cell activity to enhance anti-tumor eficacy. Understanding the role of
KIR and other receptors may allow us to exploit adoptive transfer of NK cells for therapeutic use in patients with advanced cancers.
Scientific Symposium 214
10:30 am - 12:30 pm
ROOM: 150 DEFG
Recent Advancements in the Non-Viral Delivery of Nucleic Acid Based Drugs
CO-CHAIRS: Ian MacLachlan, PhD and Olivia Merkel, PhD
SPEAKERS
Daniel G. Anderson, PhD
Combinatorial Development of Synthetic Nucleic Acid Delivery Systems
Thursday, May 16, 2013
Jianjun Cheng, PhD
a-Helical Polypeptides for Non-Viral Gene Delivery
We designed a class of helical, cationic charged polypeptides via the ring-opening polymerization of vinyl benzyl glutamic acid
N-carboxyanhydrides. These materials have unusual helical stability and display excellent cell penetrating property outperforming
TAT peptide. By controlling the chemical, morphological and self-assembly structures of these side chain charged, helical
polypeptides, we identiied a few materials that showed excellent gene/siRNA delivery eficiency in vitro and in vivo.
Thomas Knotts, PhD
Protein Structure and How it Changes in Association with Surfaces
Proteins/surface interactions are important in a variety of existing and emerging biomedical techniques and devices. It has been
known for decades that surfaces can drastically change the structure of attached or adsorbed proteins, but a priori prediction of the
changes is not currently possible. In an attempt to develop some predictive capabilities with respect to protein/surface interactions,
we have used advanced molecular simulation to test several theories as to how protein folding mechanisms and stabilities are
affected by surfaces. This talk will present many of these results, and more importantly, design heuristics that can be used to ensure
proteins of interest are stable, and functional on surfaces. Taken as a whole, the results offer compelling evidence that the rational
design of technologies involving proteins interacting with surfaces is possible.
Hamid Ghandehari, PhD
Silk-elastinlike Polymers for Matrix-mediated Gene Delivery
Final Program SALT LAKE CITY, UTAH May 15–18, 2013
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