The 15th Annual HSCI Retreat was organized by the team at Harvard Medical School as an entirely virtual event Tuesday, May 20, 2020.

The event was chaired by Jon Hoggatt (Massachusetts General Hospital) and Zuzana Tothova (Dana-Farber Cancer Institute), with keynote lectures with a focus on sickle cell gene therapy by Stuart Orkin (Dana-Farber Cancer Institute) and Erica Esrick (Boston Children’s Hospital). There were also keynote presentations by April Craft, PhD (Boston Children’s Hospital), Sarah Mojarad (University of Southern California), and Eric Olson, PhD (University of Texas Southwestern Medical Center).

Read more about this event in our blog post here, with further details being available on the HSCI website here.

HSCI Retreat 2020 Logo

HSCI Retreat 2020 Abstracts

MLL-Menin Inhibition Reverses Pre-Leukemic Progenitor Self-Renewal Induced by NPM1 Mutations and Prevents AML Development
Hannah Uckelmann,* Stephanie M. Kim, Eric M. Wong, Charles Hatton, Hugh Giovinazzo, Jayant Y. Gadrey, Andrei V. Krivtsov, Frank G. Rücker, Konstanze Döhner, Gerard M. McGeehan, Ross L. Levine, Lars Bullinger, George S. Vassiliou, and Scott A. Armstrong | View Abstract

In Vitro Characterization of the Human Segmentation Clock
Margarete Diaz Cuadros,* Daniel E. Wagner, Christoph Budjan, Alexis Hubaud, Oscar A. Tarazona, Sophia Donelly, Arthur Michaut, Ziad Al Tanoury, Kumiko Yoshioka-Kobayashi, Yusuke Niino, Ryoichiro Kageyama, Atsushi Miyawaki, Jonathan Touboul, and Olivier Pourquié | View Abstract

Genome Scale In Vivo CRISPR Screen Identifies RNLS as a Target for Beta Cell Protection in Type 1 Diabetes
Erica P. Cai ,* Yuki Ishikawa, Wei Zhang, Nayara C. Leite, Jian Li, Shurong Hou, Badr Kiaf, Jennifer Hollister-Lock, Nese Kurt Yilmaz, Celia A. Schiffer, Doug A. Melton, Stephan Kissler, and Peng Yi | View Abstract

From Bedside to the Bench: A Novel Human Homozygous IGF1R Mutation is Causal of Abnormal Skeletal Acquisition
Manisha Dixit,* Masanobu Fujimoto, Isabelle Maystadt, Andrew Dauber, Anita Rauch, Jean De Schepper, Gozde Yildirim, Yanjiao Zhang, Vivian Hwa and Shoshana Yakar | View Abstract

Organoid Maturation by Circadian Entrainment
Juan R. Alvarez-Dominguez,* Julie Donaghey, Niloofar Rasouli, Jennifer H. R. Kenty, Aharon Helman, Jocelyn Charlton, Juerg R. Straubhaar, Alexander Meissner, and Douglas A. Melton | View Abstract

Engineering Gene Therapies of Aging
Michael Florea,* Luk H. Vandenberghe, and Amy Wagers | View Abstract

A Human In Vitro Model for Type-1 Diabetes Reveals Gene Editing Targets for Immune Protection of Stem Cell-Derived Beta Cells
Elad Sintov,* Edwin Rosado-Olivieri, Nayara Leite, Adrian Veres, Henry Bushnell, David M. Harlan, Dale L. Greiner, Michael A. Brehm, and Douglas A. Melton | View Abstract

Deep Learning-Based Analysis of the Differentiation in 3D Retinal Organoids
Evgenii Kegeles,* Anton Naumov, Tatiana Perepelkina, Julia Oswald, Evgeniy Karpulevich, Pavel Volchkov, and Petr Baranov | View Abstract

Targeting Immune Checkpoint as a Novel Strategy for Anti-Aging Intervention
Yuting Tan,* Angelique Onorati, Zhixun Dou | View Abstract

Efficient, Specific and Universal Therapeutic Gene Editing of ELANE For Severe Congenital Neutropenia in Human Hematopoietic Stem Cells 
Shuquan Rao,* Josias Brito-Frazao, Qiuming Yao, Anna Victoria Serbin, Kevin Luk, Yuxuan Wu, Jing Zeng, Chunyan Ren, Anne H. Shen, Ruth Watkinson, Myriam Armant, Luca Pinello, Scot A. Wolfe, Benhur Lee, Roberto Chiarle, Akiko Shimamura, Peter Newburger, and Daniel E. Bauer | View Abstract

Transcriptional Regulation of Mitochondrial Metabolism by TIF1ƴ Drives Erythroid Progenitor Differentiation
Marlies P. Rossmann,* Karen Hoi, Victoria Chan, Julie R. Perlin, Elliott J. Hagedorn, Brian J. Abraham, Song Yang, Sejal Vyas, Partha P. Nag, Lucas B. Sullivan, Curtis R. Warren, Bilguujin Dorjsuren, Eugenia Custo Greig, Isaac Adatto, Chad A. Cowan, Stuart L. Schreiber, Richard A. Young, Marcia Haigis, and Leonard I. Zon | View Abstract

Zebrafish Chemical Compound Screen Uncovers Inducers of Skeletal Muscle Engraftment Across Species
Sahar Tavakoli,* Isaac Adatto, Sara Ashrafi Kakhki, Victoria S Chan, Haleh Fotowat, Eric Gähwiler, Margot E Manning, Kathleen A Messemer, Apoorva Rangan, Song Yang, Amy J Wagers, and Leonard I Zon | View Abstract

The Role of Extracellular Vesicles in Mesenchymal Transition and Therapeutic Resistance in Glioblastomas
Markus W. Schweiger,* Mao Li, Renata L. Fleming, Elie I. Tabet, and Bakhos A. Tannous | View Abstract

Organoid-Derived Retinal Ganglion Cells are Suitable for Cell Replacement in the Murine Retina
Julia Oswald* and Petr Baranov | View Abstract

3D-Printed ABCB5+ Sermal Stem Cells for the Treatment of Limbal Stem Cell Deficiency
Catherine A.A. Lee,* Jennifer A. Kunes, Wonhye Lee, Catherine. S. Karpova, Mahtabin R. Rozbu, Yuzuru Sasamoto, Brian J. Wilson, Bruce R. Ksander, Markus H. Frank, Seung-schick S. Yoo, and Natasha Y. Frank | View Abstract

Vascular Smooth Muscle Origin of Cold-Induced Brown Adipocytes
Farnaz Shamsi,* Matthew D. Lynes, Mary Piper, Li-Lun Ho, Tian Lian Huang, and Yu-Hua Tseng | View Abstract

Chemical Genetics Screen Identifies Vinca Alkaloids as Promoters of Platelet Formation
Arunoday Bhan,* Niharika Kareddy, Emma Forde, Bruce Furie, Glenn Merrill-Skoloff, Christian Skorik, Anjali Jha, John Atwater, Trevor Bingham, Leonard I. Zon, George Q. Daley, Andrew Frelinger, and Thorsten M. Schlaeger | View Abstract

Submitted: Jun 11 2020; Published online: Jul 27 2020