Search Results - "Potter, Rachael"

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial by Mendell, Jerry R, Sahenk, Zarife, Lehman, Kelly, Nease, Carrie, Lowes, Linda P, Miller, Natalie F, Iammarino, Megan A, Alfano, Lindsay N, Nicholl, Amanda, Al-Zaidy, Samiah, Lewis, Sarah, Church, Kathleen, Shell, Richard, Cripe, Linda H, Potter, Rachael A, Griffin, Danielle A, Pozsgai, Eric, Dugar, Ashish, Hogan, Mark, Rodino-Klapac, Louise R

“…Micro-dystrophin gene transfer shows promise for treating patients with Duchenne muscular dystrophy (DMD) using recombinant adeno-associated virus serotype…”
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Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy by Potter, Rachael A, Griffin, Danielle A, Heller, Kristin N, Peterson, Ellyn L, Clark, Emma K, Mendell, Jerry R, Rodino-Klapac, Louise R

“…Duchenne muscular dystrophy (DMD) is a rare, X-linked, fatal, degenerative neuromuscular disease caused by mutations in the gene. More than 2,000 mutations of…”
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Clinical development on the frontier: gene therapy for duchenne muscular dystrophy by Asher, Damon R, Thapa, Khampaseuth, Dharia, Sachi D, Khan, Navid, Potter, Rachael A, Rodino-Klapac, Louise R, Mendell, Jerry R

“…: The development of adeno-associated virus (AAV) vectors as safe vehicles for delivery of therapeutic genes has been a major milestone in the advancement of…”
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National Policy Index (NPI) for worker mental health and its relationship with enterprise psychosocial safety climate by Potter, Rachael E., Dollard, Maureen, Lerouge, Loic, Jain, Aditya, Leka, Stavroula, Cefaliello, Aude

“…•Mental ill health is an international public health crisis and current approaches have been ineffective.•We investigate the role of national policy in…”
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Joint ICOH-WOPS & APA-PFAW global roundtable perspectives: exploring national policy approaches for psychological health at work through the ‘National Policy Index’ lens by POTTER, Rachael E., ERTEL, Michael, DOLLARD, Maureen, LEKA, Stavroula, JAIN, Aditya, LEROUGE, Loic, HOUTMAN, Irene, AUST, Birgit, CHOI, Won-Jun, CROOKS, Nicholas, FITZGERALD, John, HASSAN, Siti Nurani, KIRK-BROWN, Andrea, MISHIBA, Takenori, SPETCH, Ashley, STOETZER, Ulrich, DIJK, Pieter VAN

“…Worker psychological health is a significant global imperative which requires national policy action and stakeholder engagement. While national policy is a…”
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The role of psychosocial safety climate on flexible work from home digital job demands and work-life conflict by PARKIN, Amy K., ZADOW, Amy J., POTTER, Rachael E., AFSHARIAN, Ali, DOLLARD, Maureen F., PIGNATA, Silvia, BAKKER, Arnold B., LUSHINGTON, Kurt

“…Due to the COVID-19 pandemic, the number of employees in flexible work from home has increased markedly along with a reliance on information communication…”
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Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy by Griffin, Danielle A, Pozsgai, Eric R, Heller, Kristin N, Potter, Rachael A, Peterson, Ellyn L, Rodino-Klapac, Louise R

“…Limb-girdle muscular dystrophy type 2D/R3 (LGMD2D/R3) is a progressive muscular dystrophy that manifests with muscle weakness, respiratory abnormalities, and…”
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Long‐term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial by Mendell, Jerry R., Sahenk, Zarife, Lehman, Kelly J., Lowes, Linda P., Reash, Natalie F., Iammarino, Megan A., Alfano, Lindsay N., Lewis, Sarah, Church, Kathleen, Shell, Richard, Potter, Rachael A., Griffin, Danielle A., Hogan, Mark, Wang, Shufang, Mason, Stefanie, Darton, Eddie, Rodino‐Klapac, Louise R.

“…Introduction/Aims Delandistrogene moxeparvovec is indicated in the United States for the treatment of ambulatory pediatric patients aged 4 through 5 years with…”
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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study SRP‐9001‐103 (ENDEAVOR) by Zaidman, Craig M., Proud, Crystal M., McDonald, Craig M., Lehman, Kelly J., Goedeker, Natalie L., Mason, Stefanie, Murphy, Alexander P., Guridi, Maitea, Wang, Shufang, Reid, Carol, Darton, Eddie, Wandel, Christoph, Lewis, Sarah, Malhotra, Jyoti, Griffin, Danielle A., Potter, Rachael A., Rodino‐Klapac, Louise R., Mendell, Jerry R.

“…Objective Delandistrogene moxeparvovec is approved in the USA for the treatment of ambulatory patients (4–5 years) with Duchenne muscular dystrophy. ENDEAVOR…”
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AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial by Mendell, Jerry R, Muntoni, Francesco, McDonald, Craig M, Mercuri, Eugenio M, Ciafaloni, Emma, Komaki, Hirofumi, Leon-Astudillo, Carmen, Nascimento, Andrés, Proud, Crystal, Schara-Schmidt, Ulrike, Veerapandiyan, Aravindhan, Zaidman, Craig M, Guridi, Maitea, Murphy, Alexander P, Reid, Carol, Wandel, Christoph, Asher, Damon R, Darton, Eddie, Mason, Stefanie, Potter, Rachael A, Singh, Teji, Zhang, Wenfei, Fontoura, Paulo, Elkins, Jacob S, Rodino-Klapac, Louise R

“…Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease caused by pathogenic variants in the DMD gene that result in the absence of…”
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Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy by Potter, Rachael A, Griffin, Danielle A, Sondergaard, Patricia C, Johnson, Ryan W, Pozsgai, Eric R, Heller, Kristin N, Peterson, Ellyn L, Lehtimäki, Kimmo K, Windish, Hillarie P, Mittal, Plavi J, Albrecht, Douglas E, Mendell, Jerry R, Rodino-Klapac, Louise R

“…Dysferlinopathies comprise a family of disorders caused by mutations in the dysferlin (DYSF) gene, leading to a progressive dystrophy characterized by chronic…”
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Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy by Mendell, Jerry R, Shieh, Perry B, McDonald, Craig M, Sahenk, Zarife, Lehman, Kelly J, Lowes, Linda P, Reash, Natalie F, Iammarino, Megan A, Alfano, Lindsay N, Sabo, Brenna, Woods, Jeremy D, Skura, Christy L, Mao, Howard C, Staudt, Loretta A, Griffin, Danielle A, Lewis, Sarah, Wang, Shufang, Potter, Rachael A, Singh, Teji, Rodino-Klapac, Louise R

“…Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy designed for targeted expression of SRP-9001 dystrophin protein, a…”
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Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74 by Potter, Rachael A., Peterson, Ellyn L., Griffin, Danielle, Cooper Olson, Grace, Lewis, Sarah, Cochran, Kyle, Mendell, Jerry R., Rodino-Klapac, Louise R.

“…Patients with pre-existing immunity to adeno-associated virus (AAV) are currently unable to receive systemic gene transfer therapies. In this nonhuman primate…”
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Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy by Potter, Rachael A., Griffin, Danielle A., Heller, Kristin N., Mendell, Jerry R., Rodino-Klapac, Louise R.

“…ABSTRACT Robust expression of shortened, functional dystrophin provided impetus to develop adeno-associated virus (AAV)–based constructs for clinical…”
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Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model by Seo, Young-Eun, Baine, Stephen H., Kempton, Amber N., Rogers, Oliver C., Lewis, Sarah, Adegboye, Kaitlin, Haile, Alex, Griffin, Danielle A., Peterson, Ellyn L., Pozsgai, Eric R., Potter, Rachael A., Rodino-Klapac, Louise R.

“…Limb-girdle muscular dystrophy (LGMD) type 2C/R5 results from mutations in the γ-sarcoglycan (SGCG) gene and is characterized by muscle weakness and…”
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Evaluation of the Lipid-binding Properties of Recombinant Dystrophin Spectrin-like Repeat Domains R1-3 by Cooper-Olson, Grace, Rodino-Klapac, Louise R, Potter, Rachael A

“…Recombinant micro-dystrophin genes are designed to treat Duchenne muscular dystrophy (DMD) by retaining dystrophin domains believed to play key functional…”
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Social antecedents and consequences of interpersonal rejection sensitivity by Butler, J. Corey, Doherty, Melissa S., Potter, Rachael M.

“…Although there is good evidence linking interpersonal rejection sensitivity to mood and anxiety disorders, less attention has been given to the social context…”
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Digital communication, health & wellbeing in universities: a double-edged sword by Potter, Rachael Elise, Zadow, Amy, Dollard, Maureen, Pignata, Silvia, Lushington, Kurt

“…We explore how digital communication relates to worker health and wellbeing within Australian universities prior to COVID-19. To investigate effects of digital…”
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Review of practice & policy strategies for managing digital communication and ICT use in Australian universities by Potter, Rachael E., Dollard, Maureen, Pignata, Silvia, Zadow, Amy, Lushington, Kurt

“…This paper explores practices and policies for managing information communication technologies (ICTs) and ensuring healthy and productive work environments in…”
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P314: Long-term safety and sustained functional benefit in patients with DMD 4 years post-treatment with delandistrogene moxeparvovec: A phase 1/2a study by O’Rourke, Erin, Mendell, Jerry R., Sahenk, Zarife, Lehman, Kelly J., Lowes, Linda P., Reash, Natalie F., Iammarino, Megan, Alfano, Lindsay, Lewis, Sarah, Church, Kathleen, Shell, Richard, Potter, Rachael A., Griffin, Danielle, Hogan, Mark J., Wang, Shufang, Mason, Stefanie, Darton, Eddie, Rodino-Klapac, Louise

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