Find Academic Supervisors

• Robotics
• Artificial Intelligence
• Robotic Manipulation of Deformable Objects
• +7 more

Carme Torras Genís is a Research Professor at the Spanish National Research Council (CSIC), affiliated with the Institute of Robotics and Industrial Informatics (IRI) in Barcelona and the Technical University of Catalonia (UPC). Her career spans over three decades, focusing on robotics, neurocomputing, and artificial intelligence with applications in healthcare and deformable object manipulation. M.Sc. in Mathematics (University of Barcelona, 1978) M.Sc. in Computer Science (University of Massachusetts, 11981) Ph.D. in Computer Science (UPC, 1984) Research Interests : Robotic manipulation of deformable objects (especially textiles) Neurocomputing and machine learning for robotic control Human-robot interaction and assistive robotics Computational topology for cloth state representation Ethics in social robotics and AI Medical applications of robotics for neuromuscular disease assessment Scientific Leadership : ERC Advanced Grant recipient (2016) IEEE and EurAI Fellow Coordinator of Horizon Europe project SoftEnable and former ERC project CLOTHILDE Editorial leadership in IEEE Transactions on Robotics and multiple journals Active in ethics committees and AI policy advisory boards Advisory Committee of Ethics in AI (Catalan Government) Vice-President of CSIC Ethics Committee Member of Royal Academy of Engineering (Spain)

Patrick J. CahillView profile

Physician Scientist

• Pediatric Spine Surgery
• Scoliosis
• Minimally Invasive Spine Surgery
• +4 more

Patrick J. Cahill, MD, is a pediatric spine specialist and the Robert M. Campbell Jr. Endowed Chair in Thoracic Insufficiency Syndrome at Children's Hospital of Philadelphia (CHOP). His clinical expertise spans disorders of the pediatric spine, scoliosis, cervical spine conditions, and minimally invasive surgical techniques. He leads the Center for Thoracic Insufficiency Syndrome, collaborating with pulmonology, anesthesia, and physical therapy teams. Academic Role: Physician Scientist Leadership: Director of the Center for Thoracic Insufficiency Syndrome Research Focus: Spinal growth modulation, 3D surgical planning, and reducing anesthesia exposure in young patients His work emphasizes fusionless treatments like magnetically expandable growing rods and Mehta casting, alongside innovations in dynamic MRI for preoperative assessment. Recent publications highlight comparative studies on spinal fusion techniques, surgical complication classifications, and multidisciplinary approaches to complex cases. Awards: Philadelphia Magazine's Top Doctors (2022), SRS Travelling Fellowship (2015) Professional Memberships: Scoliosis Research Society, North American Spine Society, Pediatric Orthopaedic Society of North America

Elliot L. DimbergView profile

Clinical Professor

• Neuromuscular Disorders
• Clinical Neurophysiology
• Peripheral Nerve Disorders
• +4 more

Dr. Elliot L. Dimberg is a neurologist specializing in neuromuscular disorders at Mayo Clinic Hospital in Jacksonville, Florida. He serves as faculty at Mayo Clinic Alix School of Medicine within the Department of Neurology, holding leadership roles including Vice Chair of the Curriculum Committee and Clerkship Sub Committee. Dr. Dimberg actively contributes to medical education through multiple committees related to student promotions, academic affairs, and residency program evaluation, while maintaining a clinical practice focused on complex neuromuscular conditions. Dr. Dimberg earned his MD from Tulane University in 2001. He completed his Neurology residency and served as Chief Resident at the University of Virginia, followed by fellowships in Clinical Neurophysiology at the University of Virginia (2006) and Neuromuscular Disease at Mayo Clinic Rochester (2008). He maintains board certification in Neurology, Clinical Neurophysiology, and Neuromuscular Medicine through the American Board of Psychiatry and Neurology. His clinical expertise spans neuromuscular junction disorders including myasthenia gravis and Lambert-Eaton Myasthenic Syndrome, peripheral neuropathies, brachial and lumbosacral plexus disorders, polyradiculopathies, motor neuron diseases, and myopathies. Dr. Dimberg integrates clinical evaluation with electrodiagnostic medicine to diagnose and manage these complex conditions. His research focuses on advancing diagnostic methodologies through electromyography techniques, genetic testing, and clinical trial participation for rare neuromuscular disorders. Dr. Dimberg's publication record demonstrates consistent contributions to neuromuscular medicine, with emphasis on diagnostic precision, genetic underpinnings of muscle disorders, and therapeutic innovations. His recent work includes clinical trials for hereditary transthyretin amyloidosis, studies on spinal muscular atrophy treatments, and investigations into immune-mediated necrotizing myopathy, reflecting his commitment to advancing both clinical practice and scientific understanding in his field. Multiple Above and Beyond Awards from Mayo Clinic in Florida (2008-2024) A.B. Baker Teacher Recognition Award from American Academy of Neurology (2013, 2021) Commitment to Education Award from Mayo Clinic Alix School of Medicine (2019) Alpha Omega Alpha Honor Society membership (2000) As an educator, Dr. Dimberg has coordinated the Residency Neuroanatomy Course and Clinical Pathological Correlation Conference for over a decade. He previously chaired the Curriculum Committee for the Adult Neurology Residency Program and currently serves in leadership roles for Mayo Clinic Alix School of Medicine's educational committees. His dedication to teaching has been recognized through numerous awards including the prestigious A.B. Baker Teacher Recognition Award. Professionally, Dr. Dimberg serves as Co-Chair of the American Association of Neuromuscular and Electrodiagnostic Medicine's EDX Lab Accreditation Committee and holds leadership positions in the American Clinical Neurophysiology Society. He contributes to developing certification exams, educational programming, and clinical guidelines for these organizations, maintaining active engagement with the broader neuromuscular medicine community.

• Clinical sciences
• Neurosciences
• Medical physiology
• +2 more

Jinhong Meng is a Senior Research Fellow at the Department of Genetics & Genomic Medicine, University College London (UCL), focusing on neuromuscular disorders and gene therapy. He earned his PhD and Bachelor’s degrees from the Fourth Military Medical University. Education: Doctor of Philosophy, Fourth Military Medical University (2000) Bachelor, Fourth Military Medical University (1995) Research Interests: Jinhong Meng’s work centers on Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA), and gene therapy techniques including CRISPR, lentiviral vectors, and antisense oligonucleotides. His studies explore immune responses to dystrophin, vascular defects in SMA, and dystrophin correction via viral and non-viral delivery systems. Publication Trends: Over the past eight years, Jinhong Meng has published extensively on DMD and SMA, with a focus on gene editing, dystrophin restoration, and cellular therapies. His collaborative work spans lentiviral vectors, foamy virus transduction, and necroptosis mechanisms in muscle degeneration. Labs & Collaborations: He works within UCL’s Genetics & Genomic Medicine Department, collaborating on projects involving dystrophic muscle engraftment, circadian signaling, and RNA editing for genetic mutations.

• Neural control of movement
• Infant development
• Motor control
• +5 more

Beth Smith, PT, DPT, PhD is an Associate Professor of Pediatrics and Biokinesiology & Physical Therapy at the University of Southern California. She directs the Infant Neuromotor Control Laboratory, where she leads research on neural control of movement during infancy and develops interventions for infants with or at risk for developmental delay. Dr. Smith's research focuses on several critical areas in pediatric development: Neural mechanisms underlying infant motor development Application of wearable sensor technology for objective movement measurement Early identification of developmental delays through quantitative analysis Evaluation of intervention effectiveness for at-risk infants Cross-cultural studies of infant development in diverse settings including rural Guatemala Her work demonstrates a strong integration of technology and clinical practice, with recent publications highlighting innovative approaches to measuring infant movement in natural environments. Dr. Smith's research on algorithmic detection of developmental disabilities using wearable sensors represents a significant advancement in early identification methods. Her studies on telehealth administration of developmental assessments have gained particular relevance following the COVID-19 pandemic, potentially expanding access to early intervention services globally. As director of the Infant Neuromotor Control Laboratory, Dr. Smith oversees a research program that bridges neuroscience, engineering, and clinical practice to improve outcomes for infants with developmental challenges. Her work has important implications for developing evidence-based interventions that can be implemented across diverse healthcare settings.

Rafael J. Yáñez-MuñozView profile

Professor

• Gene Therapy
• Cell Therapy
• Genome Editing
• +3 more

Roles and Affiliations: Rafael J. Yáñez-Muñoz is a Professor of Advanced Therapy and Director of the Centre of Gene and Cell Therapy at the Department of Biological Sciences, Royal Holloway University of London. He holds expertise in gene and cell therapy, particularly for neurodegenerative and inherited diseases such as ataxia telangiectasia and spinal muscular atrophy. Education: BSc and PhD in Biochemistry and Molecular Biology from the Autonomous University of Madrid. Previously held Lecturer positions at King’s College London and University College London. Research Interests: Focuses on developing safer gene therapy methods using episomal vectors and genome editing (e.g., CRISPR-Cas). Key areas include non-integrating lentiviral vectors, viral vector modification, and treatments for rare diseases. His lab (AGCT) explores therapies for spinal muscular atrophy, spinal injury, Parkinson’s disease, and primary immunodeficiencies. Publications and Impact: Over 85 publications, including seminal work on non-integrating lentiviral vectors and gene editing. Notable contributions to Gene Therapy as Editor-in-Chief and leadership roles in the British Society for Gene and Cell Therapy. Grants and Collaborations: Principal Investigator on projects funded by the SMA Trust, BBSRC, MRC, and others. Collaborates with institutions like Genethon (France) and Harvard University. Key initiatives include the UK SMA Research Consortium and CHASE-IT for spinal injury therapy. Awards and Roles: Trustee and Chair of Genetic Alliance UK, President of the British Society for Gene and Cell Therapy (2021–2025). Recognized for advocacy in rare disease awareness and policy, including contributions to the UK National Strategy for Rare Diseases. Advisees and Teams: Supervised numerous PhD students and postdocs, including Sahar Akbari Vala, Melika Fard, and Ellie Chilcott. Active in mentoring and lab management, with a focus on training the next generation of gene therapy researchers. Labs and Outreach: Leads the Advanced Gene and Cell Therapy (AGCT) lab, hosting annual Rare Disease Day events to raise awareness. Engages in public education through lectures, media interviews, and initiatives like the SMA Trust fundraising cycle events.

• Neurology
• Neuromuscular Diseases
• Inherited Neuropathies
• +6 more

Professor Mary M. Reilly is a distinguished academic and clinician serving as Professor of Neurology in the Department of Neuromuscular Diseases at University College London's Queen Square Institute of Neurology. She is also a consultant neurologist at the National Hospital for Neurology and Neurosurgery, Queen Square, London. Her academic appointments include: Professor of Neurology, University College London, Department of Neuromuscular Diseases Head of the Division of Clinical Neurology, UCL Queen Square Institute of Neurology Lead of the peripheral nerve clinical and research group Professor Reilly's research focuses on inherited neuropathies, with particular emphasis on Charcot-Marie-Tooth disease (CMT) and hereditary transthyretin amyloidosis (hTTRA). Her work spans the entire translational pipeline, from gene identification and pathogenetic studies through natural history studies and outcome measure development to clinical trials. She approaches her research with a patient-centered perspective, with questions arising directly from clinical encounters. Her extensive publication record demonstrates consistent contributions to understanding various forms of Charcot-Marie-Tooth disease, amyloid neuropathies, and other inherited peripheral nerve disorders. Her research employs diverse methodologies including genomic analysis, biomarker development, imaging techniques, and international collaborative studies to advance understanding and treatment of these conditions. Professor Reilly has received significant recognition for her work: Elected Fellow of the Academy of Medical Sciences (2020) Awarded Fellow of the European Academy of Neurology (2023) Past President of the Association of British Neurologists (ABN) Past President of the British Peripheral Nerve Society (BPNS) Past President of the international Peripheral Nerve Society (PNS) Current chair of the Guarantors of Brain Professor Reilly has made substantial contributions to education and mentorship in the field. As Director of Training in the inherited neuropathy consortium (INC), she developed and runs an international inherited neuropathy fellowship scheme since 2009, with 14 fellows completed. She established and directed an international fellowship program through the International Centre for Genomic Medicine in Neuromuscular Diseases (ICGNDM) between 2019-2024, connecting institutions across UK, Brazil, India, South Africa, Zambia and Turkey. She also successfully ran the MRC Neuromuscular PhD programme between 2008 and 2018, mentoring 39 students. She leads the MRC Centre for Neuromuscular Diseases Biobank London, which plays a crucial role in advancing research on rare and neuromuscular diseases through the collection and analysis of biological samples.

Elisabetta TrincheroView profile

Associate Professor

• Public Management
• Healthcare Management
• Crisis Management
• +4 more

Elisabetta Trinchero è Associate Professor at Università Bocconi's SDA Bocconi School of Management, specializing in Public Management. Since 1995, she has taught across multiple master's programs including Healthcare Management, Public Management, and Sport Management (FIFA). Ph.D. from Southern Cross University Specialized Master in Economics at Università Bocconi Second-level Specialized Master at Università Bocconi Her research focuses on strategic crisis management , risk assessment , and sustainability in public service organizations, particularly in healthcare contexts. As an AMARIS researcher, she collaborates with Southern Cross University and presents annually at AOM/IRSPM conferences. Recent publications address patient safety leadership, cross-cultural healthcare implementations, and integrated auditing frameworks. She won the 2016 BAM Best Paper Award in Public Management and Governance. Active in teaching domains: Decision Under Uncertainty, Risk Assessment, and Corporate Sustainability Develops blended-format courses in Italian on crisis management and risk management in healthcare

Zhipeng LuView profile

Associate Professor

• RNA biology
• RNA structures
• RNA machines
• +7 more

Zhipeng Lu is currently an Associate Professor of Pharmacology and Pharmaceutical Sciences at the University of Southern California (USC) School of Pharmacy. His research focuses on understanding RNA molecules and their structural complexity as a second layer of genetic instructions beyond protein encoding. He directs the Lu Lab at USC, which develops and applies novel technologies to investigate RNA structures, interactions, chemical modifications, and functions in cellular processes and animal development. Dr. Lu's research interests center on "RNA machines" in living cells, with particular emphasis on how RNA molecules fold into structures and form intermolecular interactions to execute genetic instructions. His work spans multiple dimensions of RNA biology, including RNA structure-function relationships, RNA-protein interactions, RNA modifications, and the role of RNA in human diseases such as genetic disorders and viral infections. The lab combines computational, chemical, and biological approaches to elucidate fundamental mechanisms of RNA machines, with the ultimate goal of developing new understanding and therapies targeting human diseases. Analysis of Dr. Lu's publication history reveals a strong trajectory in RNA structure and interaction mapping technologies. His work has evolved from foundational studies on RNA processing and modification to developing innovative high-throughput methods like PARIS and RISE for analyzing RNA interactomes. Recent publications focus on specific RNA systems like XIST and snoRNAs, demonstrating how his lab has moved from method development to applying these tools to solve longstanding biological questions in epigenetics and RNA therapeutics. Dr. Lu has received numerous prestigious awards recognizing his contributions to RNA research: NHGRI K99/R00 NIH Pathway to Independence Award (2017-2022) RNA Society Scaringe Award (2017) Stanford University Jump Start Award for Excellence in Research (2016-2017) Damon Runyon-Sohn Fellowship (2015-2017) His research is supported by multiple funding sources from organizations including the National Institutes of Health and other foundations. The Lu Lab is actively recruiting PhD students and postdoctoral researchers to work on several cutting-edge directions including RNA structures, interaction networks, RNA modification mechanisms, and their roles in development and disease. The lab integrates biological, chemical, and computational approaches to advance RNA biology and push forward RNA medicine. The Lu Lab at USC is a dynamic research environment focused on "RNA machines" with recent highlights including solving aspects of the orphan snoRNA problem and discovering snoRNAs that control eMet tRNA activity. The lab's vision emphasizes creative exploration of RNA biology, with researchers encouraged to pursue innovative ideas much like "wild animals running in the African savannah." Current research directions include analysis of RNA structures, interaction networks, RNA modification mechanisms, and their roles in development and disease, with applications to genetic disorders, cancers, and viral infections.

Michael SattlerView profile

Professor

• Biomolecular NMR Spectroscopy
• Structural Biology
• RNA Regulation
• +7 more

Prof. Dr. Michael Sattler is a Full Professor of Biomolecular NMR at the Technical University of Munich (TUM) and Director of the Institute of Structural Biology at Helmholtz Zentrum München. He leads the Molecular Targets & Therapeutics Center and directs the Bavarian NMR Center. His research focuses on integrative structural biology, elucidating molecular mechanisms of biological pathways through advanced NMR techniques combined with cryo-EM, SAXS, and crystallography. Key areas include RNA regulation (alternative splicing, non-coding RNAs), disease mechanisms (e.g., spinal muscular atrophy), and structure-based drug discovery for cancer and infectious diseases. Education & Career PhD in Chemistry (1995) from Goethe University Frankfurt Postdoc at Abbott Laboratories (Chicago) and EMBL Heidelberg (Group Leader, 1997–2007) Full Professor at TUM since 2007 Director roles at Helmholtz Munich and Bavarian NMR Center since 2007 Research Highlights Prof. Sattler's work has revealed structural insights into RNA:protein interactions (e.g., SF1-RNA, U2AF), SMN Tudor domain recognition, and drug discovery strategies targeting Hsp90 and viral proteases. His lab pioneered integrative structural biology approaches and established high-end NMR facilities like the 1.2 GHz spectrometer. Awards & Recognition ERC Synergy Grant (2023) Leopoldina Membership (2017) EMBO Membership (2012) Erwin Schrödinger Prize (2020) Teaching & Leadership He teaches advanced courses in NMR spectroscopy, biochemistry, and structural biology at TUM. Leads EU Horizon-funded training networks (AEGIS, RNAct) and organizes international conferences on NMR and drug discovery.

Andrea MalaspinaView profile

Professor

• Neurodegenerative Diseases
• Motor Neuron Disease (MND)
• Biomarkers Discovery
• +5 more

Andrea Malaspina serves as Honorary Professor of Neurology at Queen Mary University of London's Blizard Institute within the Faculty of Medicine and Dentistry. He holds dual clinical-academic roles as Consultant Neurologist at Barts Health and Basildon University Hospital since 2001, and Co-Director of the Barts Health MND Care and Research Centre founded in 2009. His leadership extends to the Neurodegeneration Research Group at the Blizard Institute and MND Research Directorship for DeNDRoN and CRN North-Thames. Education: MD cum Laude, University of Pavia (1991) PhD in Molecular Neurobiology, Imperial College London Professor Malaspina's research centers on neurodegenerative and neuroinflammatory pathologies with primary focus on motor neuron disease. His multidisciplinary approach integrates clinical neurology with molecular neuroscience to identify biomarkers and therapeutic targets, particularly examining phenotypic heterogeneity in ALS progression. The group employs longitudinal bio-sampling and innovative room-temperature storage techniques to track disease mechanisms across tissues and biological fluids. His publication portfolio demonstrates consistent focus on ALS biomarker discovery, with recent work emphasizing neurofilament light chain validation, tissue-fluid proteomic correlations, and phenotypic stratification. Key themes include molecular signatures of disease initiation/progression, immune-metabolic interactions, and translational applications for clinical trial design. Through the AMBRoSIA, MIROCALS, and ALS Biomarkers Study initiatives, Malaspina has established UK-wide and international collaborations for biomarker resource development. His supervisory record includes multiple PhD students and postdoctoral researchers working on neuromuscular disorders, while educational contributions span MBBS teaching, intercalated BSc programs, and the MSc Neuroscience curriculum. The Neurodegeneration Research Group maintains active partnerships with the Motor Neuron Disease Association and pharmaceutical industry, with monthly 'Biomarkers and Mechanisms' seminars and annual Blizard Neuromuscular Symposia facilitating knowledge exchange. Patient-centered research design remains integral through public involvement initiatives coordinated with MND Association.

Mandana ArbabView profile

Assistant Professor

• Neurodegeneration
• Genome Editing
• CRISPR Technology
• +2 more

Mandana Arbab is the Lodish Family Assistant Professor of Neurology at Harvard Medical School and faculty member of the Rosamund Stone Zander Translational Neuroscience Center at Boston Children's Hospital. Her research program focuses on developing precision gene editing therapies for neurodegenerative disorders. Current work employs CRISPR-based technologies to correct genetic mutations underlying conditions like spinal muscular atrophy and telomere biology disorders. Recent publications demonstrate innovations in base editing systems, including engineering novel editors with reduced off-target effects. Her 2023 Science paper established preclinical proof-of-concept for base editing in spinal muscular atrophy. Dr. Arbab's lab combines high-throughput screening, machine learning, and molecular biology to optimize therapeutic genome editing. She was awarded the NIH Pathway to Independence Award for her work advancing neurological gene therapies.

Susan IannacconeView profile

Professor

• Neuromuscular Disorders
• Pediatric Neurology
• Spinal Muscular Atrophy (SMA)
• +3 more

Dr. Susan Iannaccone is a Professor and Co-Investigator at the University of Texas Southwestern Medical Center, Department of Pediatrics, with a focus on neuromuscular disorders in children. She has held leadership roles, including Director of Pediatric Neurology (2004–2016) and Associate Director of the NIH-funded Wellstone Muscular Dystrophy Center. Her research centers on spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD), with pioneering contributions to clinical trial design and outcome measures. As site-PI for numerous trials, she has established reliable protocols for SMA and DMD, influencing global standards. She also chairs panels for Common Data Elements in neuromuscular diseases and advises patient advocacy groups like Cure SMA and the Muscular Dystrophy Association. Her work includes validating motor function outcomes for SMA through international collaborations and advancing gene therapy trials. She leads MDA-sponsored clinics, treating nearly all North Texas children diagnosed with SMA/DMD. Over 20 trainees under her mentorship now hold academic positions. Her research integrates clinical care, genetics, and translational science to improve treatment strategies for neuromuscular disorders.

Wilfried O. RossollView profile

Associate Professor

• Neurodegenerative Diseases
• Protein Aggregation
• Neuroproteomics
• +13 more

Wilfried O. Rossoll, Ph.D., is an Associate Professor of Neuroscience at Mayo Clinic in Jacksonville, Florida, and serves as Assistant Dean at the Mayo Clinic Graduate School of Biomedical Sciences. He leads the Translational Neuroproteomics Laboratory and is Director of the Multi-Omics Mass Spectrometry Core Laboratory. His research focuses on the molecular mechanisms of protein aggregation in neurodegenerative diseases such as ALS, frontotemporal dementia, and Alzheimer's disease. Ph.D., University of Vienna, Austria Master of Science, University of Vienna, Austria (with research at IMP) Dr. Rossoll's research interests center on understanding proteinopathies—diseases defined by abnormal aggregation of proteins like TDP-43 and tau. His lab uses advanced proteomics, mass spectrometry, and fluorescence microscopy to analyze neuropathological aggregates in human brain tissue and disease models. Key areas include spatial proteomics, molecular profiling of aggregates, mechanisms of protein misfolding, and identifying therapeutic modifiers of aggregation. His work bridges basic science and translational applications, aiming to develop novel therapies for neurodegenerative disorders. The recent publications highlight a strong focus on TDP-43 and tau pathology, phase transitions, stress granule dynamics, and the discovery of nuclear import receptors and other modifiers as therapeutic targets. Techniques such as proximity labeling and spatial proteomics are central to uncovering disease mechanisms and biomarkers. Standing member, Cellular and Molecular Biology of Neurodegeneration Study Section, NIH (2024–present) Associate Editor, Molecular Neurodegeneration (2020–present) Dr. Rossoll has secured multiple research grants from the National Institute on Aging and the U.S. Army, serving as Principal Investigator on projects related to TDP-43 and tau pathology, neuron-glia interactions in Alzheimer's disease, and development of CNTF receptor agonists for ALS therapy. He has mentored postdoctoral fellows and graduate students, contributing to training in neuroscience and proteomics. His laboratory collaborates extensively with the Mayo Clinic Brain Bank and neuropathology teams to validate findings in human tissues. The Translational Neuroproteomics Laboratory operates at the intersection of molecular neuroscience and proteomics, utilizing cutting-edge technologies to decode the composition and dynamics of pathological aggregates. The lab’s work is integral to Mayo Clinic’s broader research mission in neurodegenerative diseases, with a strong emphasis on translating discoveries into clinical applications.

Haiyan ZhouView profile

Professor

• Genetic Medicine
• Nucleic Acid Therapeutics
• Rare Diseases
• +7 more

Professor Haiyan Zhou serves as Professor of Genetic Medicine at University College London (UCL), affiliated with the Institute of Child Health and Department of Genetics and Genomic Medicine. She directs the UK Platform of Nucleic Acid Therapy (UPNAT Node) for rare diseases, co-leads the Therapeutic Innovation and Trials Community Domain in Genomics England, and acts as Deputy Theme Lead for Novel Therapies at the NIHR Great Ormond Street Hospital Biomedical Research Centre. Prof. Zhou earned her MD-PhD in clinical medicine and skin pharmacology from Peking Union Medical College. Her research pioneers nucleic acid therapeutics (NAT) for rare genetic disorders, focusing on RNA-based approaches including antisense oligonucleotides, siRNA, and RNA editing for neuromuscular diseases. The Zhou Lab collaborates extensively across UCL with experts in muscular dystrophies, respiratory disorders, neurological conditions, and metabolic diseases to translate experimental therapies into clinical applications for pediatric patients. Recent publications (2023-2025) reveal a dominant focus on optimizing RNA therapeutics for spinal muscular atrophy and collagen VI-related dystrophies, with key advances in allele-specific silencing, biomarker discovery, and preclinical-to-clinical translation. Her work consistently bridges molecular mechanisms with therapeutic development, emphasizing tissue-specific delivery and treatment response monitoring. Scientific recognition includes: Harrington UK Rare Disease Scholar (2021) Lea Rose Spinal Muscular Atrophy Award (2017, 2015) Young scientist award from The SMA trust (2011) President Prize for the Young Myologist of the Year (World Muscle Society, 2006) As principal investigator, Prof. Zhou secures funding from MRC, NIHR, and the Harrington Discovery Institute for RNA therapeutic development. She mentors early-career researchers through e-COST Action, Marie Skłodowska-Curie ITN, and UCL programs while supervising MSc/PhD students. Her leadership extends to directing UCL's MSc Personalized Medicine and Novel Therapies Programme and serving on multiple education committees. The Zhou Lab operates as a multidisciplinary hub within UCL's Institute of Child Health, collaborating closely with the Dubowitz Neuromuscular Centre and international consortia like the Oligonucleotide Therapeutic Society. Current initiatives focus on individualized RNA therapy for pediatric rare diseases, with active pipelines for hereditary sensory neuropathy and COL6-related dystrophies.