Guest Editors
Senthil Bhoopalan
St. Jude Children’s Research Hospital,Memphis, TN
<p>Dr. Senthil Bhoopalan is a physician-scientist with expertise in bone marrow transplantation and bone marrow failure disorders. He received his medical degree from the Government Kilpauk Medical College, Chennai, India in 2009, and subsequently moved to the US to get more rigorous research training. He obtained his PhD in Cell Biology and Molecular Genetics from the University of Maryland in 2015 under Dr. Daniel Stein’s mentorship, where he identified a novel moonlighting function of NagZ in regulating microbial biofilms. After completing a pediatric residency at the University of Nevada, he joined St. Jude Children’s Research Hospital for fellowship training in pediatric hematology and oncology. His post-doctoral research, under the mentorship of Dr. Mitchell Weiss, is focused on bone marrow failure disorders, particularly Diamond-Blackfan anemia, and using novel tools such as lentiviral vectors, Cas9, base editors, and prime editors to develop curative therapies for patients with inherited hematopoietic failures.</p>
Jonathan Yen
St. Jude Children’s Research Hospital,Memphis, TN
<p>Dr. Jonathan Yen is the director of Therapeutic Genome Engineering (TGEn) in the Department of Hematology at St. Jude Children’s Research Hospital in Memphis TN. He received his BS in biomedical engineering from Johns Hopkins University and his PhD in bioengineering at the University of Illinois at Urbana Champaign. He then continued to do his postdoctoral training in the Engineering Therapeutics group in Chemical Biology & Therapeutics at the Novartis Institutes for BioMedical Research. He worked on the application and development of non-viral delivery modalities and CRISPR-Cas9 for the treatment of sickle cell disease. After his postdoctoral training, he joined Beam Therapeutics as a scientist to develop the therapeutic application of base editors for the treatment of hemoglobinopathies. He is currently at St. Jude Children’s Research Hospital to continue to develop and apply novel genome editing tools and new delivery modalities for the treatment of hemoglobinopathies.</p><p> </p>
Collection Overview
Hematopoiesis is one of the most active systems in the human body, generating approximately one trillion cells every day. Despite its systemic complexity, advances in hematopoietic stem cell transplantation, safe lentiviral vector designs, and novel targeted genome editing techniques have made it an exciting target for clinically impactful manipulation. Over the last decade, several hundred patients have received autologous hematopoietic stem cells (HSCs) transduced with lentiviral vectors. More recently, several patients have received HSCs manipulated by genome editors such as Cas9 and zinc finger nucleases.
In this collection, we will focus on methods involved in the genetic manipulation of HSCs and related cells using viral vectors and genome editors. These genome editors would not be limited to nucleases but would also include novel technologies such as base editing, prime editing, and different genome editing modalities. This may include new delivery technologies besides electroporation. Although the exorbitant cost of developing these therapies limits access to these experimental approaches in high-income countries, we hope this Methods Collection will allow research groups worldwide to access these cutting-edge techniques.
