Gene Editing Workshop

  1. (4.00 - 4.05) Ambroise Wonkam - SickleInAfrica

    Professor of Medical Genetics, Director of GeneMap and Deputy Dean of Research the University of Cape Town. Director and Principal Investigator of the Sickle Africa Data Coordinating Center (SADaCC).
    After a MD training at University of Yaoundé I (Cameroon), he completed a thesis in Cell Biology and medical geneticist training at University of Geneva (Switzerland) and a PhD in Human Genetics (University of Cape Town, South Africa). His research interests are reflected in more than 130 peer-reviewed publications. His research focuses on Sickle Cell Disease (SCD and Genetics of hearing loss in Africa.
    Prof Wonkam has completed successfully an NIH/NHGRI funded SCD project, and a Wellcome Trust – DELTAS grant to develop capacity in human Genetic on the African continent. He has recently been granted from NIH/NHLBI 3.7m USD, to establish a Sickle Africa Data Coordinating Centre (SADaCC), to develop various studies in Tanzania, Nigeria and Ghana

  2. (4.05 - 4.10) Dr Gary H. Gibbons - NHLBI representative

    Gary H. Gibbons, M.D., is Director of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), where he oversees the third largest institute at the NIH, with an annual budget of approximately $3 billion and a staff of nearly 2,100 federal employees, contractors, and volunteers. NHLBI provides global leadership for research, training, and education programs to promote the prevention and treatment of heart, lung, and blood diseases and enhance the health of all individuals so that they can live longer and more fulfilling lives.
    Since being named Director of the NHLBI, Dr. Gibbons has enhanced the NHLBI investment in fundamental discovery science, steadily increasing the payline and number of awards for established and early stage investigators. His commitment to nurturing the next generation of scientists is manifest in expanded funding for career development and loan repayment awards as well as initiatives to facilitate the transition to independent research awards.
    Dr. Gibbons provides leadership to advance several NIH initiatives and has made many scientific contributions in the fields of vascular biology, genomic medicine, and the pathogenesis of vascular diseases. His research focuses on investigating the relationships between clinical phenotypes, behavior, molecular interactions, and social determinants on gene expression and their contribution to cardiovascular disease. Dr. Gibbons has received several patents for innovations derived from his research in the fields of vascular biology and the pathogenesis of vascular diseases.
    Dr. Gibbons earned his undergraduate degree from Princeton University in Princeton, N.J., and graduated magna cum laude from Harvard Medical School in Boston. He completed his residency and cardiology fellowship at the Harvard-affiliated Brigham and Women's Hospital in Boston. Dr. Gibbons was a member of the faculty at Stanford University in Stanford, CA, from 1990-1996, and at Harvard Medical School from 1996-1999. He joined the Morehouse School of Medicine in 1999, where he served as the founding director of the Cardiovascular Research Institute, chairperson of the Department of Physiology, and professor of physiology and medicine at the Morehouse School of Medicine, in Atlanta. While at Morehouse School of Medicine, Dr. Gibbons served as a member of the National Heart, Lung, and Blood Advisory Council from 2009-2012.
    Throughout his career, Dr. Gibbons has received numerous honors, including election to the Institute of Medicine of the National Academies of Sciences; selection as a Robert Wood Johnson Foundation Minority Faculty Development Awardee; selection as a Pew Foundation Biomedical Scholar; and recognition as an Established Investigator of the American Heart Association (AHA).

  3. (4.10 - 4.15) Emily Turner - Gates Foundation

  4. (4.15 - 4.30) Julie Makani - Gene Therapy for SCD in Africa

    Prof. Makani is a Professor in the Department of Haematology and Blood Transfusion at Muhimbili University of Health and Allied Sciences (MUHAS), the main clinical, academic and research centre in Tanzania. Tanzania has recognised sickle cell disease (SCD), as a major public health problem and it has been included as a priority condition in the national strategy for Non-Communicable Diseases in the Ministry of Health. With global partnerships, Muhimbili has developed a systematic framework for comprehensive research that is integrated into healthcare, advocacy and education. With prospective surveillance (2004 – 2016) of over 5,000 SCD patients; this is one of the largest single-center, SCD research programs in the world. Scientific themes include clinical and biomedical research (including genomics) and public health [including ethics, social/behavioral science, population health and health policy]. The aim is to use SCD as a model to establish scientific and healthcare solutions in Africa that are locally relevant and globally significant.Julie trained in Medicine (Tanzania) and Internal Medicine (UK), and completed her PhD in clinical epidemiology of SCD. She is a consultant physician at the MUHAS University Hospital, MAMC. She was a Wellcome Trust Research Fellow [Training (2003 – 2009) and Intermediate (2012 - 2017)] and Tutu Leadership Fellow (2009). She received the 2011 Royal Society Pfizer Award for her work in using anaemia in SCD as a model of translating genetic research into health benefit. She is a Fellow of Royal College of Physicians of United Kingdom and Tanzania Academy of Sciences.

  5. (4.30 - 4.45) Dr Gang Bao - Gene editing outcomes and in vivo delivery

    Dr. Gang Bao is the Foyt Family Chair Professor in the Department of Bioengineering, Rice University. He is the Associate Dean for Research and Innovation of School of Engineering, and the Director of Nanomedicine Center for Nucleoprotein Machines at Rice. Dr. Bao received his undergraduate and Master’s degrees from Shandong University in China, and his PhD from Lehigh University in the US. Dr. Bao is a Fellow of the American Association of Advancement in Science (AAAS), American Society of Mechanical Engineers (ASME), American Physical Society (APS), American Institute for Medical and Biological Engineering (AIMBE), and Biomedical Engineering Society (BMES).

    Dr. Bao’s current research is focused on the development of nanotechnology and biomolecular engineering tools for biological and disease studies, including multifunctional magnetic nanoparticles, protein tagging/targeting methods, and engineered nucleases such as CRISPR/Cas9. These approaches have been applied to the diagnosis and treatment of chronic diseases such as cancer, and the development of genome editing approaches for treating single-gene disorders.

  6. (4.45 - 4.55) Dr Siana Nkya - Understanding Human Genomics for Gene Therapy in Africa

    Dr Nkya has a background in Microbiology and Chemistry (BSc) and Molecular Biology (MSc). As part of her MSc training, she conducted research in malaria and held an internship Kemri Wellcome Trust, Mombasa, Kenya.
    Since joining Muhimbili University of Health and Allied Sciences (MUHAS) as a research scientist in 2009, she has been leading and participating in research projects related to Sickle Cell Disease (SCD). In 2015, Siana was involved in establishing a newborn screening programme for SCD at MUHAS. The primary focus of her PhD was the genetic determinants of fetal haemoglobin, during which she established a genetic database of more than 1700 individuals with SCD and undertook the first Genome Wide Association Study (GWAS) analysis of an African population in collaboration with the Wellcome Trust Sanger Institute. She held a Commonwealth Split-Site Fellowship spending one year of her PhD at King’s College London, UK.
    Siana is a lecturer in the Department of Biological Sciences at DUCE and an honorary lecturer at the Department of Biochemistry, MUHAS.
    In 2018, Siana was nominated for the Women career scientists award Tanzania by the Next Einstein Forum (NEF). She is a Fogarty Global Health Fellow (2016/2017) and an African Academy of Science affiliate (2018-2022) from Eastern Africa region. In 2018, Siana received inaugural ASH Global research award (2018-2020) and Early career fellowship (2019-2021) by the American Society of Hematology and the Organization for Women in Science for the Developing World, respectively, for conducting genomic research. Siana has also received the Emerging Global Leader Award, K43 from Fogarty, NIH.
    Siana is a co-founder of the Tanzania Society of Human Genetics (TSHG) that was initiated in 2018.

  7. (4.55 - 5.10) Matt Porteus - Gene editing for curing SCD

  8. (5.10 - 5.20) Mohammed Zahir - HSC collection and processing for Gene therapy

    Mohamed obtained his Bachelor’s degree in Biotechnology from Manipal University in India and Master’s degree in Molecular Biology and Biotechnology from The University of Groningen in The Netherlands. He is currently finalizing his PhD thesis, having completed his training in medical genetics at the Genetics Department of the University Medical Centre Groningen in The Netherlands. He works for the Innovation and Development section of the Genetics department. His research is mainly focused on using next generation sequencing to diagnose (genetic) diseases in the clinic and its implementation strategies.
    His recent works include:
    i) Developing an NGS panel that can detect translocations in acute leukemias without prior knowledge of translocation partners
    ii) Genetic variant interpretation and re-classification of single nucleotide polymorphisms, insertions and deletions (indels) and copy number variations on routine diagnostic data using targeted NGS cardiomyopathy panel
    iii) Finding functional evidence to characterize splice site mutations in experimental setting using minigene assays in patients with cardiomyopathies and
    iv) Developing targeted NGS gene panel for genetic screening of patients with Spinocerebellar ataxia.
    In addition to his research duties, he is involved in students’ project supervision and teaching activities in the field of Molecular Biology and Human Genetics.
    He is a co-founder of the Tanzania Society of Human Genetics, Executive board member of the African Society of Human Genetics, member of the Tanzania Genome Network, BOVA network, African Science Initiative, H3Africa and the Genetics Society of America. He serves as an eLife community ambassador representing Europe and Africa and a health columnist for The Citizen, Medico Press and Mahdi Health Initiative – White Coats community magazine.

  9. (5.20 - 5.30) Professor Vivien Sheehan - Rheology tests for gene-based therapy

    Dr. Sheehan is an Assistant Professor of Pediatrics in the division of Pediatric Hematology/Oncology at Baylor College of Medicine. After completing a PhD in Biochemistry, Dr. Sheehan went on to medical school at Emory University School of Medicine; there, she decided to devote her career to sickle cell patient care and research. In order to care for people of all ages with sickle cell disease (SCD), Dr. Sheehan completed an Internal Medicine/Pediatrics residency program at the University of Cincinnati; she then completed a self-designed combined Internal Medicine Hematology- Pediatric Hematology/ Oncology fellowship. Dr. Sheehan’s laboratory uses genomics to unravel the mechanisms of globin switching and the pharmacogenomics of hydroxyurea in SCD, in order to develop new fetal hemoglobin inducing agents to treat people with sickle cell disease. She collaborates with Drs. Gang Bao and Matt Porteus on CRISPR/Cas9 gene editing of hematopoietic stem cells from patients with SCD, and is developing rheology biomarkers to assess if a cure has been achieved.

  10. (5.45 - 5.55) Grace Moshi - Laboratory infrastructure for Transplantation & Gene Therapy

    Dr Grace Moshi is a haematologist, who has spent her entire career as a public hospital haematologist at university teaching hospitals. She is currently a senior consultant haematologist at the KK Women and Children’s Hospital in the Department of Pathology and Laboratory medicine as well senior consultant at the Health Sciences Authority, Blood Services Group, Transplant Support Services in Singapore, while concurrently being an honorary a visiting lecturer at the Muhimbili University of Health and Allied Sciences in Tanzania.
    Dr Moshi sits on numerous Committees and Boards; and is currently an executive committee member as well as being on the medical advisory board of the Bone Marrow Donor Program (BMDP) in Singapore, Overseas representative in Advisory Council for the Royal Australasian College of Pathologists (RCPA) of Australasia and a member of the Sickle Cell Disease Consortium in Tanzania.
    After relocating to Singapore from Australia in 2012, Dr Moshi worked as a senior consultant in haematologist at the National University Hospital as well as being in the management Board of the Tissue Engineering and Cellular therapy Laboratory at NUHS, as well as being a senior consultant at the Health Sciences Authority, Blood Services Group, Transplant Support Services in Singapore.
    Prior to moving to Singapore in 2012, Dr Moshi was a senior staff specialist in haematology at the Canberra Hospital in Australia. She has also worked as the Medical Director of the Sydney Cord Blood Bank, the Lead Consultant of the South African National Blood Service (SANBS) and haematologist at the Prince of Wales Hospital in Sydney when she was also the clinical superintendent, as well as provided rural clinical haematology services to the Grafton Base Hospital.
    Having been affiliated with the Australian diplomat corps hence the multiple relocations, Dr Moshi has had an interesting clinical portfolio, having worked in haematology at multiple other hospitals including University College Hospital (London), Hammersmith Hospital (London) and St Georges Hospital in London.

  11. (5.55 - 6.05) Yohana Mtali - HLA-Typing for transplantation

    I am a Junior scientist in the department of Hematology and blood transfusion at Muhimbili University of health and allied science. I have trained in laboratory sciences in immunology and hematology with focus on molecular diagnosis and research. I have been working as a tutorial assistant and laboratory scientist in the department of hematology, supporting several sickle projects by setting up sickle cell testing laboratory at MUHAS and provide laboratory support in testing for sickle cell diseases and research projects at MUHAS. My experience is built on training, working in the laboratory and cemented by research project conducted in my masters by research and publication studies. I recently under Sickle CHARTA program, was nominated to undergo internship training at APHP hospitals in France, the internship focused on advanced therapies for sickle cell disease, where I was able to gain knowledge and experience on laboratory aspects of hematopoietic stem cell transplant for sickle cell diseases together with setting up laboratory to support advanced therapy research and services in limited resources settings.

  12. (6.05 - 6.15) Dr Avelina Mgasa - Donor Registry & Transfusion for Transplantation & Gene Therapy

    Dr Avelina Mgasa is Medical specialist in Internal Medicine at the Ministry of Health, Tanzania. She is the Head of Blood Donor Management Department at the National Blood Transfusion Service and Adjunct lecturer at Muhimbili University of Health and Allied Sciences (MUHAS). She holds a Master of Medicine degree from MUHAS and fellowship in Capacity Building in blood transfusion from South Korea. She has trained in Human Genetics and Genomics at NIH, USA and Internship in Hematopoietic Stem Cell Transplantation in France. She is currently a PhD candidate at the Department of Molecular Biology and Biotechnology University of Dar es Salaam.

  13. (6.15 - 6.30) Dr John Tisdale - Gene Therapy for SCD

    John Tisdale received his medical degree from the Medical University of South Carolina in Charleston after obtaining his B.A. in Chemistry from the College of Charleston. He completed an internal medicine and chief residency at Vanderbilt University Medical Center in Nashville and then trained in hematology in the Hematology Branch, National Heart, Lung and Blood Institute (NHLBI), where he served as a postdoctoral fellow. He joined the Molecular and Clinical Hematology Branch of NHLBI in 1998 and is now the Chief of the Cellular and Molecular Therapeutics Branch. In 2011 the College of Charleston recognized Dr. Tisdale with the Alumni of the Year Award and the Pre-Medical Society’s Outstanding Service Award in Medicine. He was recently elected to the American Society for Clinical Investigation and is a member of the American Society of Hematology. Dr. Tisdale’s research and clinical work center on sickle cell disease. His group focuses on developing curative strategies for sickle cell disease through transplantation of allogeneic or genetically modified autologous bone marrow stem cells.

  14. (6.30 - 6.40) Dr Lucio Luzzatto - From the past to the future

    Lucio Luzzatto, MD, PhD, as an academic haematologist has always combined research work and clinical work in Ibadan, Nigeria, in London, UK, in New York, USA, as well as in his native Italy. LL has aimed consistently to the understanding in depth of both inherited and acquired blood diseases at the genetic and molecular level, for the ultimate purpose to improve their management. Currently Lucio Luzzatto is professor of Haematology at the Muhinmbili University of Health and Allied Sciences in Dar-es-Salaam Tanzania; he is involved in malaria culture work and in the Sickle-in-Africa Programme.

  15. (6.30 - 6.40) Funmi Olopade - Why Africa is the future

    Prof. Olufunmilayo is the Walter L. Palmer Distinguished Service Professor of Medicine and Human Genetics, Dean for Global Health, and Director of the Center for Innovation in Global Health at the University of Chicago. Professor Olopade develops innovative strategies for comprehensive cancer risk assessment and prevention based on evolving understanding of genetic and nongenetic factors in individual patients, with a focus on women of African ancestry across the Diaspora.
    She has received numerous honors and awards, including honorary degrees from North Central, Dominican, Bowdoin, and Princeton Universities; MacArthur Foundation Fellowship; Doris Duke Distinguished Clinical Scientist and Exceptional Mentor Award; American Cancer Society Clinical Research Professorship; Officer of the Order of the Niger Award; and the Franklin D. Roosevelt Freedom from Want Award. She is an elected member of the Association of American Physicians, National Academy of Medicine, American Academy of Arts and Sciences, and the American Philosophical Society, and currently serves on the board of directors for Susan G. Komen, Cancer IQ, Lyric Opera of Chicago, and MacArthur Foundation.

Overview (Content): 

SickleARC - Advanced Therapy (Rx), Research and Cure: Human Genomics for Gene Therapy for Africa

Extra Reading Material/Resources: 
Thursday, June 18th, 2020
3-7pm CAT, 4-8pm EAT, 9am-1pm EDT, 8am-12pm CDT, 6-10am PDT, 9pm–1am (SGT)
GotoWebinar (Please download the software at least 15 mins before session)
4 hours