Xandra Breakefield | Gene Therapy | Best Researcher Award

Xandra Breakefield | Gene Therapy | Best Researcher Award

Prof. Dr. Xandra Breakefield, Massachusetts General Hospital/Harvard Medical School, United States

Prof. Dr. Xandra Breakefield is a pioneering geneticist and neuroscientist specializing in gene therapy and extracellular vesicle research. She earned her Ph.D. from Georgetown University and trained at NIH under Nobel Laureate Dr. Marshall Nirenberg. A Harvard Medical School professor and Massachusetts General Hospital researcher, she has made groundbreaking contributions to neurogenetics and dystonia. Recognized with numerous prestigious awards 🏆, including the Mika Salpeter Lifetime Achievement Award, she ranks among the world’s top scientists. A leader in gene and cell therapy, she serves on advisory boards worldwide 🌍, shaping the future of biomedical research.

Publication Profile

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Education

Prof. Dr. Xandra Breakefield 🎓 embarked on her academic journey with an A.B. in Biology from Wilson College (1960-1965), graduating cum laude for her outstanding performance. During this period, she also enriched her knowledge at American University (1964-1965), deepening her expertise in biology 🧬. Her passion for microbial genetics led her to pursue a Ph.D. at Georgetown University (1967-1971), where she specialized in microbial genetics 🦠. This strong educational foundation set the stage for her groundbreaking contributions to the field, establishing her as a distinguished researcher in genetics and molecular biology. 🌟

Professional Experience

Prof. Dr. Xandra Breakefield 🧬 has had an illustrious career in genetics and neurology. She has been a Geneticist at Massachusetts General Hospital’s Radiology Service since 2002 and a Professor of Neurology at Harvard Medical School since 1996. Previously, she held key roles in molecular neurogenetics at Massachusetts General Hospital 🏥 and directed the Division of Molecular Neurogenetics at the Eunice Kennedy Shriver Center. Her academic journey includes faculty positions at Yale University and Georgetown University 📚. Early in her career, she trained under Nobel Laureate Dr. Marshall Nirenberg at NIH, shaping her expertise in biochemical genetics. 🌟

Awards

Prof. Dr. Xandra Breakefield 🧬 has received numerous prestigious awards for her groundbreaking contributions to neuroscience and genetics. She earned the McKnight Neuroscience Development Award (1982-1984) and two Javits Neuroscience Investigator Awards (1985-1992, 1995-2002). She was honored with an Honorary Doctorate from Wilson College (1991) and an Honorary Master’s Degree from Harvard Medical School (1996) 🎓. Recognized globally, she received the Mika Salpeter Lifetime Achievement Award (2013) and the NIH Director’s WALS Speaker Award (2017). Ranked among the Top 1000 Female Scientists in the World (2022, 2024), her work continues to shape modern molecular medicine. 🌟

Research Focus

Prof. Dr. Xandra O. Breakefield focuses on neuroscience, gene therapy, and extracellular vesicles 🧠🧬🩸. Her research explores how extracellular vesicles facilitate cell-to-cell communication and their potential for delivering gene therapies to treat neurological disorders and cancers. She also investigates glioblastoma, a highly aggressive brain tumor, using cortical organoids 🧪🔬 to study cell-state heterogeneity and intercellular transfer. Her work has significant implications for precision medicine and targeted therapies 🎯💊. As a leading expert in the field, she collaborates on innovative projects integrating genomics, bioengineering, and molecular biology to advance treatments for brain diseases.

Publication Top Notes

Prof. Stefano Rivella|Hematology and Gene Therapy| Best Researcher Award

Prof. Stefano Rivella, Hematology and Gene Therapy ,Best Researcher Award

Prof. Stefano Rivella at Children’s Hospital of Philadelphia/University of Pennsylvania,United States

PROFILE  

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🎓 Early Academic Pursuits :

Stefano Rivella’s academic journey began in Italy, where he pursued his Bachelor of Science (BSc) and Doctor of Philosophy (PhD) at the University of Pavia. His dedication to the field of genetics was evident early on, as he received Italy’s Best University Thesis award from the Italian Genetics Institute (AGI) in 1991. Following his doctoral studies, Dr. Rivella remained at the University of Pavia as a Pre and Doctoral Trainee until 1997. His postdoctoral training took him to the prestigious Memorial Sloan Kettering Cancer Center, where he deepened his expertise in gene therapy and hematology.

🧬 Professional Endeavors :

Dr. Rivella’s professional career is marked by significant contributions to genetic medicine and pediatrics. He began his faculty appointments at Weill Cornell Medical College in 2002, where he served as an Assistant Professor of Genetic Medicine in Pediatrics. His innovative work in gene therapy and pediatrics led to his promotion to Associate Professor in both the Pediatrics and Graduate School divisions by 2008.

In 2015, Dr. Rivella transitioned to the Children’s Hospital of Philadelphia (CHOP), where he became a Professor of Pediatrics. His expertise and impact in the field were further recognized when he was appointed the Kwame Ohene-Frempong Endowed Chair in Pediatric Hematology later that year. Currently, he leads the RNA Gene Therapeutics group at The Penn Institute for RNA Innovation, a role he took on in 2023. This position underscores his leadership in pioneering RNA-based therapies for genetic disorders.

🔬 Contributions and Research Focus :

Dr. Rivella’s research has focused on gene therapy, particularly in the treatment of hemoglobinopathies such as thalassemia. His work has been instrumental in the development of innovative therapeutic strategies, including the approval of the clinical trial “ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia,” which is a significant milestone in the field. This trial, sponsored by the Children’s Hospital of Philadelphia, utilizes the ALS20 vector generated in Dr. Rivella’s laboratory, reflecting his deep involvement in cutting-edge therapeutic development.

His expertise extends beyond academia, as he has served as a consultant for several major pharmaceutical companies, including Novartis, Ionis, and Merck. Dr. Rivella has also played a critical role in designing clinical trials and analyzing data for various drugs, contributing to the advancement of gene therapy and hematology on a global scale.

🏆 Accolades and Recognition :

Dr. Rivella’s contributions to the field have been widely recognized through numerous awards and honors. Early in his career, he received multiple Travel Grant Awards from prestigious organizations such as the International Society of Experimental Hematology (ISEH) and the American Society of Gene & Cell Therapy (ASGCT). Notably, in 2015, he was honored with the Sultan bin Khalifa International Thalassemia Federation Award for Innovative Medical Research, highlighting his impact on thalassemia treatment.

In 2019, Dr. Rivella was awarded the Marcel Simon Award by the International BioIron Society (IBIS), further cementing his status as a leader in the field of iron metabolism and hematology. His most recent accolade, the Outstanding Poster Award in 2024 for his work on lentiviral gene therapy, underscores his ongoing contributions to cutting-edge research in gene therapy.

🌍 Impact and Influence :

Dr. Rivella’s influence extends beyond his immediate research contributions. As a member and leader of various scientific societies, including the American Society of Hematology (ASH) and the International BioIron Society (IBIS), he has helped shape the direction of research and clinical practice in hematology and gene therapy. His role on the Scientific Advisory Boards (SAB) of several biopharmaceutical companies allows him to directly impact the development of new therapies, ensuring that innovative treatments reach patients.

🏅 Legacy and Future Contributions :

Dr. Rivella’s legacy in pediatric hematology and gene therapy is profound. Through his work, countless patients with hemoglobinopathies and other genetic disorders have new hope for effective treatments. His pioneering efforts in RNA gene therapeutics promise to revolutionize the field, offering novel approaches to previously intractable conditions.

As he continues his research and leadership at CHOP and The Penn Institute for RNA Innovation, Dr. Rivella’s future contributions are poised to further advance the field of gene therapy. His commitment to education, research, and clinical excellence ensures that his legacy will endure, inspiring future generations of scientists and clinicians dedicated to improving the lives of patients with genetic disorders.

🎓Publication 

Iron restriction in sickle cell disease: When less is more

  • Authors   :Oswaldo L. Castro; Lucia De Franceschi; Tomas Ganz; Julie Kanter; Gregory J. Kato; Sant‐Rayn Pasricha; Stefano Rivella; John C. Wood
  • Journal    :American Journal of Hematology
  • Year         :2024

Optimizing lentiviral genomic integrations to cure beta-thalassemia: The least required for success?

    • Authors :Stefano Rivella
    • Journal   :
      Molecular Therapy – Methods & Clinical Development
    • Year       : 2024

Elevated CDKN1A (P21) mediates β-thalassemia erythroid apoptosis, but its loss does not improve β-thalassemic erythropoiesis

  • Authors  :Raymond Liang; Miao Lin; Vijay Menon; Jiajing Qiu; Anagha Menon; Laura Breda; Tasleem Arif; Stefano Rivella; Saghi Ghaffari
  • Journal    :Blood Advances
  • Year         :  2023.

DNA binding to TLR9 expressed by red blood cells promotes innate immune activation and anemia

  • Authors  :L. K. Metthew Lam; Sophia Murphy; Dimitra Kokkinaki; Alessandro Venosa; Scott Sherrill-Mix; Carla Casu; Stefano Rivella; Aaron Weiner; Jeongho Park; Sunny Shin et al
  • Journal   :
    Science Translational Medicine
  • Year        :   2021

Red Blood Cells Function as DNA Sensors

  • Authors  :Metthew Lam; Sophia Murphy; Dimitra Kokkinaki; Alessandro Venosa; Scott Sherrill-Mix; Carla Casu; Stefano Rivella; Aaron Weiner; Jeongho Park; Sunny Shin et al.
  • Journal   :Preprint
  • Year        :  2021