Regenerative Therapies for Inherited Blood Disorders

The Laboratory of Regenerative Therapies for Inherited Blood Disorders, led by Dr. Andre Larochelle, is investigating novel strategies and stem cell concepts that can help advance the translational regenerative field, with a focus on inherited disorders affecting blood-forming hematopoietic stem cells (HSCs).

Andre Larochelle, M.D., Ph.D.

Images

Members of the lab in a meeting.Members of the lab in a meeting.Members of the lab in a meeting.Members of the lab in a meeting.Members of the lab in a meeting.Members of the lab in a meeting. Members of the lab in a meeting. Seven lab members standing under a tree. Three men in front of a poster. Group photo of ten members of the Regenerative Therapies for Inherited Blood Disorders lab.Group photo of members of PECASE.

Clinical Trials and Studies

Recruiting
Adult
All Genders
Accepting Healthy Volunteers
Are a healthy adult who is interested in helping people who need a bone marrow transplant? This procedure is often associated with life-threatening reactions, such as graft-versus-host disease (GHVD). This study will test a technique that uses peripheral blood instead of bone marrow. With this technique, they can transplant donor stem cells and remove lymphocytes, which are the white blood cells from donor bone marrow that can cause GHVD. To participate, you must be between 18 and 60 years old. This study is located at the NIH Clinical Center in Bethesda, Maryland.
Recruiting
All Ages
All Genders
Not Accepting Healthy Volunteers
Do you or your child have Fanconi anemia with lower blood cell counts? This study aims to see if a new drug called eltrombopag will help people with this condition. Participants must be at least 2 years old. The study is located at the NIH Clinical Center in Bethesda, Maryland.

Meet the Team

Andre Larochelle, M.D., Ph.D.

Andre Larochelle, M.D., Ph.D.

Senior Investigator

With a career spanning over three decades, Dr. Larochelle brings a unique blend of expertise in both fundamental hematopoiesis and clinical hematology. His journey began in 1992 when he delved into the intricacies of hematopoietic stem and progenitor cell (HSPC) regulation, culminating in a Ph.D. from the University of Toronto in 1996. Recognizing the transformative potential of bridging basic science with clinical practice, he accelerated his medical education, completing a three-year MD program at McMaster University in 1999. His medical training continued at the Mayo Clinic, followed by a Clinical Hematology Fellowship at the NIH, where he became a board-certified clinical hematologist and stem cell transplant specialist. Dr. Larochelle chose to extend his tenure at the NIH, initially as a post-doctoral trainee and later as a Staff Clinician and Associate Clinical Investigator within the Hematology Branch of the NHLBI. During this period, he spearheaded pivotal projects focused on the mobilization, genetic modification, and expansion of HSPCs for clinical applications in cell and gene therapy. In 2016, he was appointed as a tenure-track investigator, and by 2024, he was promoted to senior investigator. He continues to pioneer innovative strategies in this capacity, utilizing gene therapy and editing technologies to advance regenerative therapies for inherited disorders affecting HSPCs. His contributions to the field have been widely recognized, both through numerous peer-reviewed publications and invitations to speak at prestigious events. He is an active member of the American Society of Hematology and the American Society of Gene and Cell Therapy. He was honored with the 2016 Presidential Early Career Awards for Scientists and Engineers (PECASE), the U.S. Government's highest accolade for early-stage science and engineering professionals.

Alumni

Patali Shikhi Cheruku, M.D.

Postbaccalaureate Trainee
2014 - 2016

Hai Cheng, M.D.

Postdoctoral Trainee
2014 - 2016

Luigi Alvarado, Ph.D.

Postdoctoral Trainee
2014 - 2018

Fatima Sancheznieto (previously Juan Pablo Ruiz), Ph.D.

Pre-Doc Graduate Student
2014 - 2018

Heather Huntsman, Ph.D.

Postdoctoral Trainee
2014 - 2018

Jianfei Fu, M.D., Ph.D.

Postdoctoral Trainee
2014 - 2019

Joseph Panos, M.D.

Summer Trainee
2015 - 2015

Kevin Bachrach, M.D.

Summer Trainee
2015 - 2015

Neranjan DeSilva, M.Sc.

Summer Trainee
2015 - 2015

Ishan Asokan, M.D.

MRSP Medical Student
2015 - 2016

Roop Dutta, M.D.

MRSP Medical Student
2015 - 2016

Michael Drakopoulos, M.D.

Summer Trainee
2016 - 2016

Noah Zachary Laird, Ph.D.

Summer Trainee
2015 - 2016

Kacey Bui Gunther, B.Sc.

Postbaccalaureate Trainee
2016 - 2018

Daisuke Araki, M.D.

Clinical Trainee
2017 - 2019

Beth Staskal, B.Sc.

Summer Trainee
2017 - 2017

Husam Qanash, Ph.D.

Pre-Doc Graduate Student
2017 - 2020

Hanan Bloomer, B.Sc.

Postbaccalaureate Trainee
2018 - 2020

Waleed Hakami, Ph.D.

Pre-Doc Graduate Student
2018 - 2022

Yongqin Li, Ph.D.

Postdoctoral Trainee
2018 - 2023

Nebojša Skorupan, M.D.

Clinical Trainee
2019 - 2021

Shing Gao

Summer Trainee
2022 - 2022

Anirudh Rao, B.Sc.

MRSP Medical Student
2022 - 2023

Emily Pollack

Summer Trainee
2023 - 2023