LGMD "Entrevista en el punto de mira de la investigación"

INVESTIGADOR LGMD: Rita Perlingeiro

Afiliación: University of Minnesota

Función o cargo:  Professor of Medicine

¿Qué educación y formación ha recibido para llegar a su puesto actual?

PhD in Physiology at the University of Campinas (UNICAMP) in Sao Paulo, Brazil, followed by postdoctoral studies at the Whitehead Institute at MIT, Cambridge-MA.

¿Qué le llevó a dedicarse a la investigación y, en particular, al estudio de la distrofia muscular?

I wanted to be a scientist since I was a little kid. Throughout my career, the main motivation has been the desire to make in impact in people’s lives through our research.

¿Qué temas está estudiando?

The main topic of research in our laboratory is the development of stem cell-based therapies for muscular dystrophies, and this includes a better understanding of the molecular signature of pluripotent stem cell-derived myogenic progenitors and engrafted stem cells, the signals that allow for these cells to home to diseased muscles upon systemic transplantation and promote muscle regeneration, gene correction of LGMD patient-specific induced pluripotent stem (iPS) cells for assessing the potential of an autologous stem cell-based therapy, and optimization studies for the scalability, manufacturing and safety of pluripotent stem cell-derived myogenic progenitors for therapeutic applications.

¿Cómo ayudará su trabajo a los pacientes? ¿Tiene un carácter más científico o podría convertirse en un tratamiento para LGMD o MD en general?

When we began these studies in 2004, they were purely basic science, but as we advanced our research over the years, we feel we are now much closer to translating this approach into a potential treatment for LGMD and other MDs. We had a pre-IND meeting with the FDA in 2017 to align our short- and long-term goals.

¿Qué le gustaría que los pacientes y otras personas interesadas en la LGMD supieran sobre la investigación (sus propios proyectos y sobre el campo en general)?

There are many obstacles and many unknowns in the road of bringing a new therapeutic treatment for MD testing, but our lab and others are working fearlessly towards this goal.

¿Qué le inspira a seguir trabajando en este campo?

The possibility that our work may make a difference in the lives of patients with MDs.

¿Cómo pueden los pacientes animarle y ayudarle en su trabajo?

The strength and hope of MD patients constantly give us significant encouragement. Over the years, patients have helped our work through tissue donation and financial support and we are grateful for their support.

 

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* * * To learn more about Limb Girdle Muscular Dystrophy (LGMD), please visit our website at: https://lgmd-info.org