Innovations in Science
Development of next-generation therapies for Duchenne muscular dystrophy
Duchenne muscular dystrophy is a severe muscle disorder caused by genetic abnormalities which affect the normal production of a functional molecule, called dystrophin. One way to remediate this situation is to introduce correct copies of dystrophin into the affected muscles using a biological carrier. This technique is named gene replacement. Among the numerous agents developed in medical research, Adeno-associated viral vectors (AAV) are found to be the most efficient gene carriers. However, these vectors present a limited cargo capacity and are unable to transport large genes like dystrophin. Here, we present a new method in which we split the dystrophin gene into multiple fragments transportable by AAV. Once in the cell, these fragments are reassembled into a functional protein using molecules called inteins. This method can be used to replace any gene larger than what an AAV vector can accommodate and could have a broad application.
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Grantee: Hichem Tasfaout, PhD
Grant type: Development Grant
Award total: $210,000.00
Institution: University of Washington
Country: USA
Development of optimized transgenes and capsids for treating LGMD2A/R1
This proposal aims to optimize CAPN3 transgenes and capsids for gene therapy for a form of limb girdle muscular dystrophy, LGMD R1/2A.
Partial funding for this grant comes from the Coalition to Cure Calpain.
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Grantee: Melissa Spencer, PhD
Grant type: Research Grant
Award total: $300,000.00
Institution: The Regents of the University of California, Los Angeles
Country: USA
Stem Cell Targeting in Duchenne Muscular Dystrophy
Correcting genetic defects within the endogenous muscle SCs could provide a long-term therapy for muscle disorders with potential to repair a patient’s muscles over his/her lifetime. Although a number of studies have now shown muscle SC targeting is feasible using AAV, the efficiency is low, and it is still not known if these studies efficiently targeted quiescent muscle SCs and if there are any associated toxicities after transduction that prevent their ability to continuously restore dystrophin after multiple rounds of injury. This work will perform a careful evaluation of AAV toxicities in stem cells (Aim 1) and functionality after targeting (Aim 2) using conventional (AAV9) and novel AAV stem cell targeting variants.
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Grantee: April Pyle, PhD
Grant type: Research Grant
Award total: $300,000.00
Institution: The Regents of the University of California, Los Angeles
Country: USA
The mechanism, synergies, and therapeutic horizon of quercetin in DM and C9-ALS
Our project aims to investigate the potential of a natural dietary substance, quercetin, for treating the most common cause of adult-onset muscular dystrophy (myotonic dystrophy) and a type of amyotrophic lateral sclerosis (C9-ALS). These conditions are caused by expansion of specific repetitive DNA sequence, resulting in detrimental changes to both RNA and proteins. Currently, there are no effective FDA approved treatments available for this disease that target their root cause. Preliminary findings from myotonic dystrophy suggest quercetin can mitigate these harmful effects at the RNA level, showing potential as a novel and promising treatment. This proposed project has three main goals: firstly, to understand how quercetin interacts with and affects the disease-causing repeat expansions; secondly, to find the best combination of quercetin with other treatments to improve its effectiveness; and thirdly, to assess the therapeutic potential of quercetin for two other repeat expansion disease, myotonic dystrophy type 2 and C9-ALS. Our work is guided by the belief that understanding and targeting the fundamental causes of these diseases can lead to breakthroughs in treatment. By rigorously testing these compounds in both cell and animal models, we aim to pave the way for clinical trials, moving a step closer to delivering effective treatments for patients grappling with these challenging genetic diseases.
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Grantee: Subodh Mishra, PhD
Grant type: Development Grant
Award total: $207,916.00
Institution: Research Foundation of SUNY - University at Albany
Country: USA
microRNA as biomarkers for phenoconversion in pre familial ALS
Amyotrophic lateral sclerosis (ALS) is severe neurodegenerative condition of the human motor neuron system. Some cases of ALS are associated with genetic mutations, but it is not known whether, or at what age, individuals carrying these mutations will manifest the symptoms of the disease, as they remain without symptoms for many years. If there were blood tests of patient condition, they could have been tremendously helpful in answering these questions. However, today there are no such blood tests.The Hornstein lab at Weizmann Institute of Science innovate blood tests for ALS. We use groundbreaking technologies for measuring RNA in the bloodstream, as new means to predict disease severity. The RNA markers that we offer to develop in this program will take our technology one step further and may provide a new panel of non-invasive tools for disease characterization, enhance basic research of disease and facilitate drug development.
Partial funding for this grant comes from AFM Telethon.
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Grantee: Eran Hornstein MD, PhD
Grant type: Research Grant
Award total: $300,000
Institution: Weizmann Institute of Science
Country: Israel
Addressing Immunotoxicities to AAV Gene Therapy for DMD and Other Disorders
Adeno associated virus (AAV) gene therapies provide transformative and often lifesaving treatments for otherwise intractable disorders. As such, they are becoming a new treatment paradigm with FDA approvals increasing and scores of new AAV-based therapies rapidly being developed. For decades, AAV viral vectors have been known to elicit efficacy limiting innate immune responses. Nevertheless, their clinical safety record remained impressive at relatively low doses. More recently however, doses have risen dramatically to achieve therapeutic benefit after systemic administration. Most prevalently alongside high-dose systemic administration, serious and sometimes fatal immunotoxicities have emerged as models to predict and adjust for them have failed. With the recent FDA approval of Elevidys to treat Duchenne muscular dystrophy, and its likely to be expanded label, the largest patient population ever will soon receive AAV. To predict and address AAV immunotoxicities, we have innovated a highly sensitive novel assay measuring innate immune signals associated with key unsafe responses to AAV genomes. Here, we propose to 1) establish the power of our assay to predict immunotoxicities in patients administered AAV at Stanford, 2) to build out our assay’s optionality for multifaceted interrogation of licensed products, and 3) to use our assay to immunologically de-risk candidate AAV split intein dystrophin sequences that may surpass the therapeutic benefit of Elevidys.
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Grantee: Bradley Hamilton, PhD
Grant type: Development Grant
Award total: $210,000.00
Institution: Stanford University School of Medicine
Country: USA
Single-cell Multiomic analysis of cellular and epigenetic dynamics in EDMD
Laminopathies are a large group of human degenerative disease, of which the most common involve heart and muscles. Despite varying in terms of symptoms these disorders are caused by defects in two closely related protein called Lamin A and C that are normally associated with nuclear membrane of cells. How the loss of these proteins leads to the development of muscular degeneration or affects the heart is not completely understood. It is known however that affected cells display different degrees of abnormalities in their nuclear shape. Overall, destabilization of nuclear membrane could impact how DNA is packed and accessed which, in turn, influences which genes are expressed. These proteins are found in most cell types in the human body and so, in principle, each of these cell types could be impacted. Here we focus on their potential role in muscle loss and on how the mutual interaction of different cell types are altered in this context. To do so we propose to use a new strategy that combines different layers of information: gene expression, imaging and DNA accessibility and is capable of study all the different cell type simultaneously. This new approach will enable us to and correlate the different nuclear abnormalities with their potential correlation with gene expression to better understand the steps that leads from the genetic mutation to the outcome of the disease.
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Grantee: Lorenzo Giordani, PhD
Grant type: Research Grant
Award total: $299,573.00
Institution: Sorbonne Université-Inserm UMRS974
Country: France
High-fidelity dual RNA-targeting CRISPR systems for the C9ORF72 repeat RNAs
An abnormal expansion of a GGGGCC hexanucleotide repeat in the C9ORF72 gene is the most common genetic cause of amyotrophic lateral sclerosis (ALS). To date, three non-exclusive mechanisms have been proposed to explain the pathogenicity of the hexanucleotide repeat expansion, including a gain-of-function toxicity originating from the production of sense and antisense repeat-containing RNAs from the C9ORF72 gene. Given the evidence in support of a role of these repeat RNAs in C9ORF72-related ALS (C9-ALS), strategies for silencing their production hold immense promise for the disorder; however, there remains a major need for precision approaches capable of targeting both mutant repeat RNA species to effectively mitigate the neurotoxicity of the hexanucleotide repeat. One emerging platform with the potential to enable this are RNA-targeting CRISPR effector proteins, a class of enzymes that includes the Cas13 family of proteins and Cas7-11. Utilizing these two effector proteins, we now aim to develop high-fidelity CRISPR systems to silence both the sense and antisense repeat RNAs. Thus, by utilizing an innovative technology whose capabilities are uniquely suited for C9-ALS, we will develop new and permanently effective targeted gene therapies for this devastating and currently incurable disorder.
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Grantee: Thomas Gaj, PhD
Grant type: Research Grant
Award total: $300,000.00
Institution: The Board of Trustees of the University of Illinois Urbana-Champaign
Country: USA
HEALEY ALS Platform Trial
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease for which there is no cure yet. Recent discoveries in ALS research are generating a rich pipeline of promising drugs that await testing in clinical trials, and new hope that effective treatments may be within reach.
The Healey & AMG Center at Massachusetts General Hospital, in partnership with the Northeast ALS Consortium, launched the HEALEY ALS Platform Trial in 2020. The study team focused on developing a patient-centric trial to 1) support and empower the ALS community; 2) accelerate drug development by testing multiple drugs simultaneously; and 3) advance scientific knowledge through testing of novel biomarkers that can provide answers more rapidly than traditional endpoints.
Now that we have established an efficient and robust platform trial for people living with ALS, we are at a turning point to capitalize upon what we have learned from the trial so far, share our data and resources with the ALS and other disease communities, and adapt the trial for even greater efficiency.
With MDA’s continued support, we propose to 1) accelerate ALS scientific knowledge and drug development through biomarker research; 2) enhance patient outreach and recruitment through collaboration with MDA Care Centers and MOVR; and 3) engage with the FDA to adapt the trial based on previous experience and share our learnings with the community. Robust collaboration with MDA will be critical to achieve such goals.
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Grantee: Sabrina Paganoni MD, PhD
Grant type: Clinical Research Grant
Award total: $500,000.00
Institution: Massachusetts General Hospital (Mass General)
Country: USA
Prime editing system for the in vivo correction of muscular dystrophy mutations
Limb girdle muscular dystrophies (LGMD) typically impact adolescents who experience progressive loss of skeletal muscle function and in many cases cardiac muscle degeneration that leads to premature death. Currently, there are no therapeutics to effectively treat LGMD. This project combines stem cell, nanoparticle, mouse model and genome editing technologies in translational studies to develop a corrective gene repair therapy for a common mutation in individuals with the R7 subtype of LGMD. The technologies developed will provide a pathway to the clinic for LGMDR7 as well as a platform to correct other gene mutations that cause the diversity of LGMD and other muscular dystrophies.
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Grantee: Scot Wolfe, PhD
Grant type: Research Grant
Award total: $300,000.00
Institution: University of Massachusetts Chan Medical School
Country: USA
Novel type of monogenic diabetes in patients with Welander distal myopathy
Hitherto unknown clinical findings show that patients with Welander distal myopathy (WDM), a dominantly inherited muscular disease, may be at increased risk of getting diabetes of an atypical form. WDM is caused by a mutation in TIA1, a key link to failure of insulin-producing cells in diabetes. AIM We will determine whether WDM patients have increased risk of diabetes and explore the causes and possibilities for treatment and prevention. METHODOLOGY Patients will drink a concentrated sugar solution on a single occasion. Blood samples are then collected on different time points over 2 hours for analyses of blood sugar and hormones. The results will be compared to relatives without WDM. Our PRELIMINARY RESULTS show that >30% of WDM patients have diagnosed diabetes, which is far more than expected and previously unknown. This overrepresentation of diabetes suggests that WDM and diabetes may have similar causes. The WDM TIA1 mutation causes loss of insulin production, promoting diabetes, an event prevented by the diabetes drug liraglutide. SIGNIFICANCE The diabetes in WDM patients may be a novel form of diabetes caused by their TIA1 mutation. The events causing diabetes in WDM patients may be possible to treat or prevent by already approved antidiabetic drugs (liraglutide). Beneficial effects of such drugs against muscular disease in humans have been recently reported. Additionally, the FDA has approved use of such a drug against muscular dystrophy.
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Grantee: Ake Sjoholm MD, PhD
Grant type: Research Grant
Award total: $94,414.00
Institution: Region Gavleborg
Country: Sweden
Early neuromesodermal defects underlie SMA pathology
Even in the late-onset most common neurodegenerative diseases, where neuropathologies only manifest years after birth, often after decades, unanticipated neurodevelopmental alterations are starting to be revealed. The main objective of this study is to investigate whether early developmental defects constitute the basis of the pathology affecting motor neurons and muscle cells in SMA. We will use complex patient-derived 3D in vitro models and a commonly used mouse model of SMA to interrogate the cellular and molecular basis of such developmental defects. We are confident that this study, if successful, will uncover that neuromuscular degeneration in severe forms of SMA is programmed during the early development of the spinal cord and will therefore open up new avenues for developing combinatorial therapies for SMA.
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Grantee: Natalia Rodriguez-Muela, PhD
Grant type: Research Grant
Award total: $298,988.53
Institution: German Center for Neurodegenerative Diseases (Deutsches Zentrum für Neurodegenerative Erkrankungen - DZNE)
Country: Germany
The role of cholesterol metabolism abnormalities in Coenzyme Q deficiency
Coenzyme Q is a lipid present in all cell membranes and involved in many biological functions, including energy production in the mitochondria. Defects in the making of CoQ cause Primary CoQ deficiencies, which often manifest in infancy or childhood with involvement of several organs, including kidney, brain, and heart. We studied CoQ-deficient kidney disease for many years and showed that defect in energy production is not the only mechanism responsible for kidney failure. In this application, we propose a novel mechanism of disease responsible for brain and heart diseases in CoQ deficiency. Since CoQ is produced through the same pathway that produces cholesterol and the two branches are co-regulated, we aim to establish the role of alterations in cholesterol metabolism in the demise of brain and heart in the contest of CoQ deficiency, using a novel mouse model that partially reproduces the human disease, and heart and brain cells derived from patients skin fibroblasts. The ultimate goal is to discover potential therapeutic targets for CoQ deficiency and other diseases where low CoQ has been found. In fact, patients often do not respond to CoQ supplementation because CoQ does not accumulate effectvely in tissues.
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Grantee: Catarina Quinzii, MD
Grant type: Research Grant
Award total: $300,000.00
Institution: Columbia University Irving Medical Center
Country: USA
Targeting overexpressed transcription co-regulators to suppress SBMA
Kennedy’s disease is caused by a mutation in the androgen receptor (AR). AR is the receptor of testosterone, the sex male hormone, and of note, only males show symptoms. AR is a transcription factor, which means that it controls what the cells need to produce (express) or not to stay healthy. It does so by interacting with other proteins called transcriptional coactivators. The mutation leads to the production of a protein with altered functions; some are lost, and others are enhanced. We have collected evidence that by modulating the interaction with two specific AR partners that are increased during the disease, we can reduce the aberrant AR activity while preserving its normal function. We propose to investigate i) how the enhancement of these two factors in muscle contributes to motor neuron and muscle pathology; ii) whether one of these two factors modifies the disease protein; and iii) we propose to move our approach to the clinics using nanoparticles driven to muscle to inhibit these two factors and test whether this could be a successful therapeutic strategy for patients. Our experimental approach is based on strong preliminary data and is relevant for two reasons: it will increase our knowledge of the mechanisms governing AR function in pathophysiological contexts, and it may allow us to move our tools to phase I clinical trial.
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Grantee: Maria Pennuto, PhD
Grant type: Research Grant
Award total: $300,000.00
Institution: University of Padua (Università degli Studi di Padova)
Country: Italy
Investigating mechanistic drivers of MuSK autoimmunity
Myasthenia gravis (MG) is a rare neurological disease characterized by muscle weakness. This disease is caused by antibodies that bind to specific proteins expressed on muscle cells, blocking the communication between nerves and muscles. In a subset of patients, such antibodies recognize a protein called muscle-specific tyrosine kinase (MuSK). In MuSK MG, muscle weakness is typically severe, and most patients experience difficulty breathing, swallowing, and talking. Importantly, these symptoms respond well to rituximab, a medication that reduces the number of white blood cells producing anti-MuSK antibodies. Following rituximab, most patients show no symptoms for several months. But despite such striking improvements, the disease eventually reappears, requiring hospitalization and additional treatments. Researchers have discovered the precise mechanism through which anti-MuSK antibodies cause muscle weakness. These advances notwithstanding, there remain unknowns about the cause and persistence of the disease, hindering a definitive cure. Our project will investigate which factors contribute to the chronic course of MuSK MG. First, we will study the cells that cause the disease and how they mature to produce the antibodies. Second, we will examine whether gut bacteria can mimic MuSK and increase the production of anti-MuSK antibodies, a process known as “molecular mimicry”. Our research is designed to provide new insights that could lead to better treatments for MuSK MG.
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Grantee: Gianvito Masi, MD
Grant type: Development Grant
Award total: $207,815.00
Institution: Yale University
Country: USA
Therapeutic effects of Antisense Oligonucleotide for Treatment of VCP Disease
VCP disease is a devastating complex disease for which there is no treatment. The disease is associated with muscle wasting and weakness, Paget’s disease of the bone, frontotemporal dementia, and amyotrophic lateral sclerosis. Studies in many laboratories have shown a gain of function mechanism of the VCP gene as the cause of the disease. We plan to reduce VCP activity to normal values with antisense oligonucleotides (ASOs) in order to correct the disease-related pathology in muscle, and nerve. ASO technology has emerged as a powerful strategy for the treatment of genetic disorders such as spinal muscular atrophy. ASOs are short, synthetic single-stranded DNA sequences designed to target RNA and once bound, the target RNA becomes degraded. In collaboration with Ionis Pharmaceuticals, ASOs targeting human VCP mRNA were designed and studied in our laboratory. The ASOS have shown approximately 50% knockdown in quadriceps and diaphragms of mice. The long-term objective is to conduct a clinical trial of VCP targeting ASOs in patients with VCP disease. Stem cells made from patients with VCP disease have been differentiated successfully in our lab into myoblasts and neurons (muscle and nerve cells) which will be used for our translational studies. We plan to perform detailed studies of different doses of the most promising ASO2 to determine the optimum dose and impact on VCP pathology and improvement in muscle strength and function of the mice.
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Grantee: Virginia Kimonis MD, MRCP
Grant type: Research Grant
Award total: $300,000.00
Institution: The Regents of the University of California (Irvine)
Country: USA
Notch Pathway modulation in Satellite Cells of two novel mdx models
Duchenne Muscular Dystrophy patients present fast muscular degeneration due to the lack of muscle dystrophin. Therefore, research has focused on rescue dystrophin expression. However, unique cases of Golden Retriever Muscular Dystrophy dogs that had a normal lifespan and, more recently, rare Duchenne patients with a mild course raise the question: Is it possible to significantly extend motor function without dystrophin? Our findings strongly support this idea, and we found a common biochemical pathway among these unique dogs and human patients. This pathway is known as the Notch pathway, a master regulator of muscle regeneration. We investigated our findings using animal models and found beneficial effects. To fast-track this project to a novel DMD therapy, we propose investigating the molecular mechanisms of Notch modulation in dystrophic muscles and testing a pharmacological strategy. Since our approach bypasses dystrophin up-regulation, it could benefit all DMD patients independently of their specific mutation and other forms of muscular dystrophies.
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Grantee: Felipe de Souza Leite, PhD
Grant type: Development Grant
Award total: $210,000.00
Institution: Boston Children's Hospital
Country: USA
Aberrant mt-mRNA folding as mediator of mitochondrial encephalomyopaties
Mitochondrial diseases are disorders that affect how our cells produce energy. They are most common in children and frequently lead to problems in the brain and heart. We want to study two specific genes that are linked to these diseases: ATP6 and ATP8. Such genes provide the instructions for making the energetic system of the cell. However, the exact way these genes work is not fully understood. We want to use new techniques to study how the genes are used to make proteins. We believe that a special process might be responsible for making sure the proteins are produced in the right amount. We also believe that understanding such process would explain the disease. Knowing how the disease works will help us find new ways to treat these conditions in the future.
Partial funding for this grant comes from AFM Telethon.
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Grantee: Michele Brischigliaro, PhD
Grant type: Development Grant
Award total: $210,000
Institution: Miller School of Medicine of the University of Miami
Country: USA
Defining the Protective Effects of Estrogen in FSHD
Facioscapulohumeral Muscular Dystrophy (FSHD) is a highly prevalent muscular dystrophy with no known cure. Clinical studies in patients with FSHD indicate females are typically less affected and more frequently asymptomatic compared to males. We have found similar sex-differences in a mouse model of FSHD, with females developing less severe pathology compared to males. Higher levels of circulating estrogens in females have been proposed as a potential mechanism for this difference, but if and how estrogens mitigate disease severity is poorly understood. Estrogen plays a critical role in the regulation of skeletal muscle mass, strength, and endurance through multiple mechanisms, and may interfere with the negative effects of double homeobox 4 (DUX4) protein expression -- the proposed cause of FSHD. Therefore, in Aim 1 and 2 we will investigate the effects of estrogen on DUX4 pathological activity and muscle health and function in a mouse model of FSHD. In Aim 3, we will investigate the protective effect of estrogen on the health and function of human “mini muscles” generated from muscle precursor cells isolated from FSHD and Healthy, male and female donors. We anticipate these experiments will: 1) elucidate the mechanisms contributing to less severe disease in females; and 2) provide insight into the potential utility to estrogen signaling as a therapeutic strategy to reduce symptoms and improve muscle health in males and females with FSHD.
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Grantee: Adam Bittel, PhD
Grant type: Development Grant
Award total: $210,000.00
Institution: Children's Research Institute (CNMC)
Country: USA
Muscular dystrophy therapy via angiogenesis
Duchenne muscular dystrophy (DMD) is caused by mutations in a gene called dystrophin. Dystrophin maintains the structure and function of muscle fibers throughout the body and prevents damage caused by muscle contractions. Currently, there is no definitive treatment for DMD patients, and current therapies focus on prolonging survival and improving quality of life. This proposal attempts to reduce muscle fiber damage by promoting angiogenesis and vascular recovery, which may help in the development of new treatments for DMD through increased vascular density in blood-deficient dystrophic muscle.
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Grantee: Atsushi Asakura, PhD
Grant type: Research Grant
Award total: $300,000.00
Institution: Regents of the University of Minnesota - Twin Cities
Country: USA
New Pathways of Protein Homeostasis in ALS
The study is aimed at elucidating a new mechanism of sensing misfolded proteins and regulating their synthesis related to ALS. The outcome could have important implications for understanding protein homeostasis as a common theme of the disease. The findings will not only provide novel insights into the pathogenic mechanisms in ALS but also identify new therapeutic targets for this devastating disease.
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Grantee: Jeffrey Rothstein MD, PhD
Grant type: Restricted Research Grant
Award total: $76,005.00
Institution: Johns Hopkins University School of Medicine
Country: USA
Paired Prime Editors to treat Friedreich’s Ataxia
Prime editing (PE), a novel type of gene editing, represents a promising therapeutic approach for FA because it addresses its root cause, the removal of the GAA expansions in the FXN gene. The advantage of gene editing approaches over gene therapy for FA is that, upon removal of the GAA repeats, the edited FXN gene will still be under the control of its natural regulator, thus alleviating concerns about the overexpression of frataxin and associated toxicity. This multi-disciplinary team of investigators will test several novel PE tools, comparing their efficacy and specificity. The aim is to demonstrate robust and specific GAA repeat removal from the FXN gene in human FA cells. One approach, PRIME–Del, uses an editing tool in which only one strand of the DNA is cut. This may reduce efficiency but will also reduce unwanted edits in the DNA (off targets). PE-Cas9-based deletion and repair (PEDAR) uses an editing tool that cuts both strands of the DNA, which may prove to be more efficient but has the safety liability of inversions and deletions in the DNA. One liability of PE is that it requires a machinery that is very large and doesn’t fit in AAV, the viral system used to deliver genetic medicines to tissues. Therefore, the investigators have devised a system in which two halves (split editors) of the gene editing machinery are delivered separately. By comparing efficiency and specificity of these different approaches the team will be able to identify an editing tool with the optimal combination of high efficiency of editing and reduced rate of off-targets in the genome.
Co-funded with Friedreich's Ataxia Research Alliance.
Grantee: Jonathan Watts, PhD
Grant type: Research Grant
Award total: $125,000.00
Institution: University of Massachusetts Chan Medical School
Country: USA
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