Burden of Bleeding Episodes Among Persons With Hemophilia B

Burden of Bleeding Episodes Among Persons With Hemophilia B

AWARDED/PRESENTED: 2013
GRANT/PROGRAM:
Bleeding Disorders Conference
Peer Support/Outreach/Integration Models
RESEARCHERS:
Xiaoli Niu, Jiat-Ling Poon, Judith Baker, Megan Ullman, Mimi Lou, Marion Koerper, Kathy Parish, Randall Curtis, Mike B. Nichol
In vivo Selection of Hematopoietic Stem Cells that are Genetically-Modified to Express Platelet-FVIII for Hemophilia A Gene Therapy

In vivo Selection of Hematopoietic Stem Cells that are Genetically-Modified to Express Platelet-FVIII for Hemophilia A Gene Therapy

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Gene Therapy
Platelets
Hemophilia A (Factor VIII/F8)
Author(s):
Yingyu Chen

The goal of Dr. Chen's research is to examine a method for selectively expanding hematopoietic stem cells expressing the factor VIII transgene. She will also examine the immune consequences of this approach, based on the idea that gene transfer in platelets evades immune recognition. This research has the potential to elicit important clues to developing an approach for gene therapy of hemophilia A and hemophilia A with inhibitors.

Dr. Chen earned a PhD in hematology from Fujian Medical University in China. She already has more than 27 papers published in the Chinese medical literature. Her research in hemophilia and gene therapy will be under the mentorship of Dr. Qizhen Shi, MD, PhD, Associate Investigator at the Blood Research Institute and Assistant Professor of Pediatric Hematology at the Medical College of Wisconsin.

Stepping Up and Reaching Out to the Community

Stepping Up and Reaching Out to the Community

Year: 2013
Grants:
Bleeding Disorders Conference
Peer Support/Outreach/Integration Models
Author(s):
Edward J. Kuebler, Madeline Cantini

Objective:

Who better to understand and help advocate for the bleeding disorders community than those who live it every day? Step Up Reach Out (SURO) was created to help foster the next generation of leaders in the bleeding disorders community.

Method:

SURO is an international leadership program designed to help build tomorrow's leaders in the bleeding disorders community. The program was created by the University of Texas Health Science Center, Gulf States Hemophilia and Thrombophilia Center (UTHS) in 2007 with support from Bayer HealthCare.

SURO brings together young people (18-24 years old) from around the world for learning, personal growth and collaboration. This one-year program consists of two sessions of leadership training, activities focused on developing communications skills, and individual and group projects. During the time between the two sessions, participants are asked to identify an area in their communities for which they would like to step up and define an action plan, putting into practice the skills they have acquired. They continue to learn from and support one another through the SURO Alumni Network.

Summary:

To date, the SURO program has trained nearly 100 individuals from more than 20 countries, ranging from the US to Mexico, India and beyond. SURO alumni have come out of the program ready to put their action plans to the test, developing programming to support their local community needs.

Conclusions:

SURO helps participants build self-esteem, develop concrete thinking abilities and make decisions that will help make them become leaders in their own right.