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THE HERITABLE DISORDERS OF CONNECTIVE TISSUE SUNDAY, JULY 10

6:30-9:00: Registration

MONDAY, JULY 11
7th floor Auditorium Shriners Hospital for Children
8:45-9:00: Introduction

Session I:
Therapies for Skin Disorders (Chair: Leena Bruckner-
Tuderman)
9:00-9:25:
Jakub Tolar “Hematogenous cells for skin extracellular matrix
deficiency”
Leena Bruckner-Tuderman “DEB mouse: wound healing, cancer
and therapeutic perspectives”
10:00-10:15: Vitali Alexeev “Development of protein therapeutics for Junctional
10:20-10:50: Coffee Break
10:50-11:05: Tracy Adair-Kirk “Dox-controllable, keratinocyte-targeted
expression of human laminin 2 rescues laminin 2 KO mice and generates a model of junctional epidermolysis bullosa” 11:10-11:35: Jouni Uitto “Pseudoxanthoma Elasticum—the paradigm of ectopic
mineralization disorders: towards treatment and cure” 11:40-12:05: Anne De Paepe “New genes and molecular pathways in the
12:10-12:30: General Discussion
Lunch: 12:30-1:30 in the 7th floor Auditorium foyer, Play Deck, or Board Room
Session II: New Targets for Treating Fibrosis (Chair: Leena Bruckner-
Tuderman)
1:30-1:55:
Elizabeth Gerber “New treatment strategies for scleroderma
derived from mouse models of Stiff Skin Syndrome”
Dean Sheppard “TGF activation by integrins”
Diane Shelton “Fibrosis in canine muscular dystrophies and other
inherited myopathies”
Olga Igoucheva “Disease-site targeting and application of adult
stem cells for heritable skin and muscle disorders”
Gerhard Sengle “Microenvironmental regulation by fibrillin-1”
Poster Session, Wine and Cheese Reception in the Auditorium foyer
TUESDAY, JULY 12
7th floor Auditorium Shriners Hospital for Children
Session III: Treating Bone Disorders (Chair: Hans Peter Bächinger)
9:00-9:25: Hans Peter Bächinger “The collagen folding machinery and
David Russell “Eliminating collagen mutations in Osteogenesis
Imperfecta stem cells”
10:00-10:15: Cathleen Raggio “Comparison of Rankl inhibition and alendronate
treatment in a growing mouse model of Osteogenesis Imperfecta” 10:20-10:50: Coffee Break
10:50-11:05: Elena Pokidysheva “The use of minoxidil, a translational inhibitor
for lysyl hydroxylases, as a drug for treatment of recessive Osteogenesis Imperfecta” 11:10-11:35: Gerard Karsenty “Serotonin regulation of bone mass”
11:40-11:55: Sherrill Adams “Type III collagen: an essential component of the
12:00-12:30: General Discussion
Lunch: 12:30-1:30 in the 7th floor Auditorium foyer, Play Deck, or Board Room
Session IV: Novel Therapeutic Approaches for the Chondrodysplasias
(Chair: William Horton)
Kazuwa Nakao “Translational science of the CNP/GC-B/cGMP
system for achondroplasia”
Bill Horton “New strategies for targeting FGFR3 in achondroplasia”
Eileen Shore “Fibrodysplasia Ossificans Progressiva—a genetic
disorder of ectopic endochondral ossification”
Linda Sandell “The cartilage collagen N-propeptide, PIIBNP,
inhibits angiogenesis and kills tumors”
Jacqueline Hecht “CHOP is essential for mutant COMP retention
and apoptosis in chondrocytes in an inducible transgenic mouse
model”
Banquet: 6:30-9:30 at the Heathman Hotel Welcome from Carolyn Levering, CEO and President, and Josephine
Grima, Vice President, of the National Marfan Foundation
Featured Speaker: David L. Rimoin, M.D., Ph.D., Steven Spielberg Chair
and Distinguished Professor of Pediatrics, Cedars-Sinai Medical Center,
David Geffen School of Medicine at UCLA

WEDNESDAY, JULY 13
7th floor Auditorium Shriners Hospital for Children

Session V: Therapies for Vascular Disorders (Chair: Hal Dietz)
9:00-9:25:
Hal Dietz “Found in translation: new insights regarding the
pathogenesis of Marfan syndrome and related disorders”
Fransiska Malfait “Clinical, biochemical and molecular overview of
the Ehlers-Danlos syndrome vascular type”
9:50-10:05: Julie De Backer “New therapeutic challenges in vascular EDS and
10:10-10:40: Coffee Break
10:40-10:55: Wanfen Xiong “Doxycycline attenuates TGF- activation and
aneurysm rupture through its MMP-2 inhibition in Marfan syndrome” 11:00-11:25: Francesco Ramirez “Genetic dissection of bone and vascular
11:30-12:00: General Discussion
Lunch: 12:00-1:00 in the 7th floor Auditorium foyer, Play Deck, or Board Room
Session VI: New Targets for Treating Marfan Syndrome and Related
Disorders (Chair: Lynn Sakai)
1:00-1:25:
Valerie Cormier-Daire “Understanding
ADAMTS/ADAMTSL/Fibrillin functions by studying the acromelic
dysplasia group”
Dan Rifkin “Multiple functions of LTBPs in Marfan-related
phenotypes; a new challenge for therapy”
Lynn Sakai “Microfibril fragmentation and BMP signaling in Marfan
syndrome”
Jason Cook “Genetic dissection of vascular pathology in Marfan
syndrome”
Hiromi Yanagisawa “Dual role of fibulin-5 in prevention of pelvic
organ prolapse”
Alison Frand “The fibrillin-like Fbn-1 protein of C.elegans regulates
molting cycles”

THURSDAY, JULY 14
7th floor Auditorium Shriners Hospital for Children
Session VII: Hot Topics (Chair: Peter Byers)
Peter Byers “Clinical and molecular heterogeneity of recessive
Osteogenesis Imperfecta”
Elena Makareeva “Chaperoning osteogenesis: new protein folding
paradigms and their implications for Osteogenesis Imperfecta”
Sergei Boudko “Structural and biophysical studies of the mutation
in cyclophilin B that causes hyperelastosis cutis in the American
Quarter Horse”
10:00-10:30 Coffee Break
Session VIII: Summary and Recommendations (Chair: Peter Byers; Co-
Chairs: Leena Bruckner-Tuderman, Hans Peter Bächinger, Bill Horton, Hal
Dietz, and Lynn Sakai)
10:30-10:55: Peter Byers “New Directions: where is the compass?”
11:00-12:00: General Discussion

Source: http://www.shcc.org/HDCT%202011%20Meeting/Final%20Program.pdf