Training Fellows

Peggie Chien, B.S.

Peggie Chien, B.S.

Dean's Scholar Prize

Prior Training
University of Rochester, B.S. Cell and Developmental Biology

Program
MBIDP, Cell and Developmental Biology

Mentor
April Pyle, Ph.D. (Fall Rotation)

Skeletal muscle progenitor cells (SMPCs) differentiated from human pluripotent stem cells (hPSCs) are difficult to expand while maintaining their stemness. This makes it difficult to use them for transplantation in cell therapies for muscular dystrophies. Recently, the Pyle lab has established a GFP reporter hPSC line that reflects the expression of PAX7, a critical factor for muscle cell stemness. My project focuses on using SMPCs derived from this reporter cell line to screen for small molecules that can support the growth and maintenance of PAX7+ progenitor cells.

Nathaniel Elia, B.S.

Nathaniel Elia, B.S.

MCIP IDP T32 Fellow

Prior Training
UC Davis, B.S. in Neurobiology, Physiology, and Behavior

Program
Molecular, Cellular & Integrative Physiology

Mentor
Stephen Cannon, M.D., Ph.D.

Our lab studies the pathogenic basis of skeletal muscle excitability disorders due to ion channel mutations, with an emphasis on a group of channelopathies resulting in attacks of weakness known as periodic paralysis. My research centers around one such potassium channelopathy, known as Andersen-Tawil Syndrome, which affects both skeletal and cardiac muscle excitability. My work aims to understand the physiological basis which triggers such attacks, and characterize the phenotypic manifestation of the disease in both skeletal and cardiac muscle.

Michael Emami, B.S.

Michael Emami, B.S.

MBI T32 Fellow

Prior Training
UC Irvine, B.S.

Program
MBIDP, Cell and Developmental Biology

Mentor
Melissa Spencer, Ph.D.

I am interested in using CRISPR/Cas9 editing as a therapeutic strategy for Duchenne muscular dystrophy. I am also focusing on alternative editing platforms such as CRISPR/CpfI.

Josh Lee, MS

Josh Lee, M.S.

Muscle Cell Biology T32 Fellow

Prior Training
UC Irvine, MS

Program
Molecular Biology Institute

Mentor
Yibin Wang, Ph.D.

Dystrophin deficiency in Duchenne Muscular Dystrophy (DMD) causes the loss of muscle membrane integrity and increases muscle cells to stress-induced damages, leading to progressive wasting of both skeletal and cardiac muscles. Due to heart muscle loss, most DMD patients develop dilated cardiomyopathy (DCM), along with fibrosis and cardiac arrhythmias in their early to middle teens and die of congestive heart failure only a few years post onset of DCM symptoms. Currently, intense efforts have been made to use human induced pluripotent stem cell derived cardiomyocytes (hiPSC-CM) in therapy, disease modeling, and drug screening in hopes of finding better therapeutic options for DMD patients. However, these in vitro derived myocytes are incredibly immature and very limited in possible utilizations. In a novel approach to mature hiPSC-CMs in vitro, I combined alternative splicing modulation with a bioengineered platform. WIth this, I hope to better groom hiPSC-CMs for practical uses, while shedding light on the gene regulatory mechanisms that govern muscle maturation, which thus has proved to be elusive.

Amanda Lin, B.S.

Amanda Lin, B.S.

Muscle Cell Biology T32 Fellow

Prior Training
UCLA, B.S.

Program
Molecular, Cellular & Integrative Physiology

Mentor
Andrea Hevener, Ph.D.

Duchenne muscular dystrophy (DMD) is a severe and progressive muscle wasting disorder caused by the absence of the protein dystrophin. DMD is characterized by fragile dystrophin-deficient muscle fibers and Ca2+-driven muscle degenerative and inflammatory pathways including the rapid synthesis of heat shock proteins (HSPs), a family of chaperone proteins. Our lab and collaborators have shown that genetic overexpression of an inducible HSP, HSP72, enhances metabolic function and mitochondrial health in muscle and slows the progression of muscle dysfunction in mouse models of DMD. HSP72 is also the most prevalent HSP induced under cellular stress including acute nutrient excess and endurance exercise. As exercise is a primary strategy to help maintain muscle strength and function in DMD patients, identifying the molecular actions of HSP72 in muscle is critical to understanding the exercise response and these findings can lead to improvements in quality of life for DMD patients.

Kholoud Saleh

Kholoud Saleh, B.S.

Qatar Foundation Fellow

Prior Training
UCLA, Research Scholar
Qatar University, B.S. in Biological Sciences

Program
Molecular, Cellular & Integrative Physiology

Mentor
April Pyle, Ph.D.

Skeletal muscle is one of few organs in the human body that has its own stem cell niche capable of repopulating muscle fibers upon injury. Satellite cells (SCs), which lie between the sarcolemma of muscle fiber and basal lamina, form an adult stem cell source for muscle repair. In Duchenne Muscular Dystrophy (DMD), a devastating genetic disease characterized by muscle wasting, the SCs are rendered dysfunctional following continuous muscle insult. The migratory nature of these progenitors is of particular interest in the settings where systemic delivery in cell-based therapy is desired. My research will examine the migratory potential of skeletal muscle progenitor cells (SMPCs) and investigate their functionality in skeletal muscle niche.

Cynthia Shu, B.S.

Cynthia Shu, B.S.

Prior Training
UC Irvine, B.S. Developmental and Cell Biology

Program
Molecular Biology Interdepartmental PhD Program (MBIDP)
Home area: Cell and Developmental Biology

Mentor
Rachelle Crosbie-Watson, PhD

My research focuses on developing therapeutic approaches to treat various forms of muscular dystrophy. In collaboration with the Molecular Screened Shared Resource (MSSR) at UCLA, I am developing a high throughput screen to identify small molecules that can upregulate sarcospan expression. Overexpression of sarcospan in the Duchenne muscular dystrophy mouse model is shown to improve muscle health and function by stabilizing the muscle membrane. Thus, identifying small molecules that can regulate sarcospan expression may lead to new treatments for the muscular dystrophies.

Courtney Young, M.S.

Courtney Young, M.S.

UCLA Dissertation Year Fellowship, 2017-present

Prior Training
University College London, M.S.,
Johns Hopkins University, B.S.

Program
MBIDP, Cell and Developmental Biology

Mentor
Melissa Spencer, Ph.D.

We are developing a CRISPR/Cas9-mediated gene editing platform to correct the reading frame for up to 60% of Duchenne muscular dystrophy patients. We are applying our platform to DMD human induced pluripotent stem cells and can restore dystrophin protein and function after differentiation to cardiac and skeletal muscle. Furthermore we are currently developing in vivo strategies for our gene editing approach.

Florian Barthélémy, Ph.D.

Florian Barthélémy, Ph.D.

Prior Training
Aix Marseille University/UMRS-910
(Marseille, France) Ph.D. degree

Assessment of new drugs to promote exon skipping for Duchenne Muscular Dystrophy. This project is to extend preclinical translational data in support of the therapeutic development path in relation to exon skipping for DMD. We have identified drugs that target RyR regulated Ca+ flux (RyR-antags) and synergize with AON in promoting human DMD exon 51 and mouse exon 23 skipping and rescue of an internally truncated, but partially functional dystrophin protein (Kendall et al, 2012). Now, we have secured industry partnerships that enable us to test proprietary compounds targeting these same pathways, but which have potential for even better therapeutic window(s) and commercialization.

Elizabeth Gibbs, Ph.D.

Elizabeth Gibbs, Ph.D.

Prior Training
University of Michigan, M.S., Ph.D.

Mentor
Rachelle Crosbie-Watson, Ph.D.

Muscle weakness in Duchenne muscular dystrophy is caused by the loss of a protein called dystrophin, which serves to protect the muscle cell membrane during contraction. I study the interaction of protein complexes that help stabilize the cell membrane, and how these complexes can be used to prevent membrane damage in Duchenne muscular dystrophy.

Michael Hicks

Michael Hicks, Ph.D.

Eli and Edythe Broad Stem Cell Research Center Fellowship

Prior Training
Arizona State University

Mentor
April Pyle, Ph.D.

My research uses human pluripotent stem cells (HPSCs) to make skeletal muscle progenitor cells (SMPCs) as a means to repair and replace damaged muscle fibers. Dr. Pyle’s laboratory is at the forefront of using these cells to deliver dystrophin protein to mouse models of Duchenne Muscular Dystrophy in xenograft transplantations. I seek to test and improve upon the best differentiation protocols in the scientific and preclinical field to create SMPCs with robust myogenic potential and serve the role of an endogenous adult muscle stem cell. Much of my work is currently characterizing the muscle derived from HPSCs, maturing these cells via modeling the muscle stem cell niche, and identifying small molecules and growth factors to further drive their myogenesis.

Kristen Stearns-Reider, Ph.D., P.T.

Kristen Stearns-Reider, Ph.D., P.T.

Prior Training
UC Irvine, B.S., Biological Sciences,
UCSF, M.S., Physical Therapy,
USC, Ph.D., Biokinesiology

Mentor
Rachelle Crosbie-Watson, Ph.D.

My current research is focused on the role of skeletal muscle extracellular matrix (ECM) in muscle pathology associated with Duchenne muscular dystrophy (DMD). I study ECM biophysical and biochemical alterations that may contribute to DMD muscle pathology, and how the ECM is altered following gene and exon skipping therapies for DMD. Given the importance of the muscle microenvironment in directing resident cell function, research into ECM alterations in DMD will enhance our understanding of DMD muscle pathology and may provide insight into new therapies that facilitate and enhance the formation of new skeletal muscle.

Haibin Xi

Haibin Xi, Ph.D.

Prior Training
University of Miami, Ph.D.

Mentor
April Pyle, PhD

During the progression of DMD, the constant damage of patients' skeletal muscle results in abnormal activation of the resident skeletal muscle stem cells (satellite cells, SCs), which leads to their exhaustion and eventually the loss of muscle regeneration. Therefore, to cure DMD, it is imperative to replenish patients with SCs or skeletal muscle progenitor cells (SMPCs) to support their long-term muscle maintenance. In this regard, human pluripotent stem cells (hPSCs) are a superior source for obtaining SMPCs due to their remarkable self-renewal and differentiation capabilities. My project is focusing on devloping an efficient and defined protocol to differentiate hPSCs to generate SMPCs following developmental myogenesis and understand the indentity of the in vitro-derived SMPCs. The ultimate goal is to use hPSC-derived SMPCs in cell transplantation therapy to cure DMD.

Zhenqi Zhou, Ph.D.

Zhenqi Zhou, Ph.D.

Prior Training
Wellstone Fellow

Mentor
Andrea Hevener, Ph.D.

I am interested in mitochondrial dynamics and quality control in regulating skeletal muscle metabolism and the pathophysiology of muscular dystrophy. Early diagnosis of Duchenne muscular dystrophy (DMD) could enable early treatment that delay the symptoms and improve the quality of life. My current research focuses on detecting novel serum biomarkers to assess disease progression and response to therapies in mouse muscle dystrophy models and exploring the physiological role of these biomarkers in vivo.

Joana Capote

Joana Capote, Ph.D.

Brian McMorran, MS

Brian McMorran, Ph.D.

Ekaterina Mokhonova, Ph.D.

Ekaterina Mokhonova, Ph.D.

Michelle S. Parvatiyar, Ph.D.

Michelle S. Parvatiyar, Ph.D.

Derek Wang, B.S.

Derek Wang, Ph.D.