Search Results - "Sahenk, Zarife"

AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1 by Ozes, Burcak, Myers, Morgan, Moss, Kyle, Mckinney, Jennifer, Ridgley, Alicia, Chen, Lei, Bai, Shasha, Abrams, Charles K., Freidin, Mona M., Mendell, Jerry R., Sahenk, Zarife

“…X-linked Charcot-Marie-Tooth neuropathy (CMTX) is caused by mutations in the gene encoding Gap Junction Protein Beta-1 (GJB1)/Connexin32 (Cx32) in Schwann…”
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PUMILIO hyperactivity drives premature aging of Norad -deficient mice by Kopp, Florian, Elguindy, Mahmoud M, Yalvac, Mehmet E, Zhang, He, Chen, Beibei, Gillett, Frank A, Lee, Sungyul, Sivakumar, Sushama, Yu, Hongtao, Xie, Yang, Mishra, Prashant, Sahenk, Zarife, Mendell, Joshua T

“…Although numerous long noncoding RNAs (lncRNAs) have been identified, our understanding of their roles in mammalian physiology remains limited. Here, we…”
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Eteplirsen for the treatment of Duchenne muscular dystrophy by Mendell, Jerry R., Rodino-Klapac, Louise R., Sahenk, Zarife, Roush, Kandice, Bird, Loren, Lowes, Linda P., Alfano, Lindsay, Gomez, Ann Maria, Lewis, Sarah, Kota, Janaiah, Malik, Vinod, Shontz, Kim, Walker, Christopher M., Flanigan, Kevin M., Corridore, Marco, Kean, John R., Allen, Hugh D., Shilling, Chris, Melia, Kathleen R., Sazani, Peter, Saoud, Jay B., Kaye, Edward M.

“…Objective In prior open‐label studies, eteplirsen, a phosphorodiamidate morpholino oligomer, enabled dystrophin production in Duchenne muscular dystrophy (DMD)…”
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A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy by Mendell, Jerry R, Sahenk, Zarife, Malik, Vinod, Gomez, Ana M, Flanigan, Kevin M, Lowes, Linda P, Alfano, Lindsay N, Berry, Katherine, Meadows, Eric, Lewis, Sarah, Braun, Lyndsey, Shontz, Kim, Rouhana, Maria, Clark, Kelly Reed, Rosales, Xiomara Q, Al-Zaidy, Samiah, Govoni, Alessandra, Rodino-Klapac, Louise R, Hogan, Mark J, Kaspar, Brian K

“…Becker muscular dystrophy (BMD) is a variant of dystrophin deficiency resulting from DMD gene mutations. Phenotype is variable with loss of ambulation in late…”
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Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes by Mendell, Jerry R., Sahenk, Zarife, Al-Zaidy, Samiah, Rodino-Klapac, Louise R., Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Miller, Natalie, Yalvac, Mehmet, Dvorchik, Igor, Moore-Clingenpeel, Melissa, Flanigan, Kevin M., Church, Kathleen, Shontz, Kim, Curry, Choumpree, Lewis, Sarah, McColly, Markus, Hogan, Mark J., Kaspar, Brian K.

“…Sporadic inclusion body myositis, a variant of inflammatory myopathy, has features distinct from polymyositis/dermatomyositis. The disease affects men more…”
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AAV1.NT-3 gene therapy in the SOD1KO mouse model of accelerated sarcopenia by Tong, Lingying, Ozes, Burcak, Moss, Kyle, Myers, Morgan, Ridgley, Alicia, Sahenk, Zarife

“…Sarcopenia, an age-related loss of muscle mass, is a critical factor that affects the health of the older adults. The SOD1KO mouse is deficient of Cu/Zn…”
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Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors by Haidet, Amanda M, Rizo, Liza, Handy, Chalonda, Umapathi, Priya, Eagle, Amy, Shilling, Chris, Boue, Daniel, Martin, Paul T, Sahenk, Zarife, Mendell, Jerry R, Kaspar, Brian K

“…Increasing the size and strength of muscles represents a promising therapeutic strategy for musculoskeletal disorders, and interest has focused on myostatin, a…”
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Gentamicin-induced readthrough of stop codons in duchenne muscular dystrophy by Malik, Vinod, Rodino-Klapac, Louise R., Viollet, Laurence, Wall, Cheryl, King, Wendy, Al-Dahhak, Roula, Lewis, Sarah, Shilling, Christopher J., Kota, Janaiah, Serrano-Munuera, Carmen, Hayes, John, Mahan, John D., Campbell, Katherine J., Banwell, Brenda, Dasouki, Majed, Watts, Victoria, Sivakumar, Kumaraswamy, Bien-Willner, Ricardo, Flanigan, Kevin M., Sahenk, Zarife, Barohn, Richard J., Walker, Christopher M., Mendell, Jerry R.

“…Objective The objective of this study was to establish the feasibility of long‐term gentamicin dosing to achieve stop codon readthrough and produce full‐length…”
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Gene therapy for muscular dystrophy: Lessons learned and path forward by Mendell, Jerry R., Rodino-Klapac, Louise, Sahenk, Zarife, Malik, Vinod, Kaspar, Brian K., Walker, Christopher M., Clark, K. Reed

“…► Molecular therapeutic approaches to treat muscular dystrophies are discussed. ► Exon skipping targets pre-mRNA allowing one or more exons to be omitted. ►…”
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Mutation of FIG4 causes a rapidly progressive, asymmetric neuronal degeneration by Zhang, Xuebao, Chow, Clement Y., Sahenk, Zarife, Shy, Michael E., Meisler, Miriam H., Li, Jun

“…Recessive Charcot-Marie-Tooth disease type-4J (CMT4J) and its animal model, the pale tremor mouse (plt), are caused by mutations of the FIG4 gene encoding a…”
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Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model by Rodino-Klapac, Louise R, Janssen, Paul M L, Shontz, Kimberly M, Canan, Benjamin, Montgomery, Chrystal L, Griffin, Danielle, Heller, Kristin, Schmelzer, Leah, Handy, Chalonda, Clark, K Reed, Sahenk, Zarife, Mendell, Jerry R, Kaspar, Brian K

“…Pharmacologic strategies have provided modest improvement in the devastating muscle-wasting disease, Duchenne muscular dystrophy (DMD). Pre-clinical gene…”
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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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Vascular Delivery of rAAVrh74.MCK.GALGT2 to the Gastrocnemius Muscle of the Rhesus Macaque Stimulates the Expression of Dystrophin and Laminin α2 Surrogates by Chicoine, Louis G., Rodino-Klapac, Louise R., Shao, Guohong, Xu, Rui, Bremer, William G., Camboni, Marybeth, Golden, Bethannie, Montgomery, Chrystal L., Shontz, Kimberly, Heller, Kristin N., Griffin, Danielle A., Lewis, Sarah, Coley, Brian D., Walker, Christopher M., Clark, K. Reed, Sahenk, Zarife, Mendell, Jerry R., Martin, Paul T.

“…Overexpression of GALGT2 in skeletal muscle can stimulate the glycosylation of α dystroglycan and the upregulation of normally synaptic dystroglycan-binding…”
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Follistatin gene delivery enhances muscle growth and strength in nonhuman primates by Kota, Janaiah, Handy, Chalonda R, Haidet, Amanda M, Montgomery, Chrystal L, Eagle, Amy, Rodino-Klapac, Louise R, Tucker, Danielle, Shilling, Christopher J, Therlfall, Walter R, Walker, Christopher M, Weisbrode, Steven E, Janssen, Paul M L, Clark, K Reed, Sahenk, Zarife, Mendell, Jerry R, Kaspar, Brian K

“…Antagonists of myostatin, a blood-borne negative regulator of muscle growth produced in muscle cells, have shown considerable promise for enhancing muscle mass…”
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Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1 by Sahenk, Zarife, Ozes, Burcak, Murrey, Darren, Myers, Morgan, Moss, Kyle, Yalvac, Mehmet E., Ridgley, Alicia, Chen, Lei, Mendell, Jerry R.

“…Limb girdle muscular dystrophy (LGMD) 2A/R1, caused by mutations in the CAPN3 gene and CAPN3 loss of function, is known to play a role in disease…”
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Dystrophin glycoprotein complex dysfunction: A regulatory link between muscular dystrophy and cancer cachexia by Acharyya, Swarnali, Butchbach, Matthew E.R., Sahenk, Zarife, Wang, Huating, Saji, Motoyasu, Carathers, Micheal, Ringel, Matthew D., Skipworth, Richard J.E., Fearon, Kenneth C.H., Hollingsworth, Michael A., Muscarella, Peter, Burghes, Arthur H.M., Rafael-Fortney, Jill A., Guttridge, Denis C.

“…Cachexia contributes to nearly a third of all cancer deaths, yet the mechanisms underlying skeletal muscle wasting in this syndrome remain poorly defined. We…”
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Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD by Wallace, Lindsay M, Saad, Nizar Y, Pyne, Nettie K, Fowler, Allison M, Eidahl, Jocelyn O, Domire, Jacqueline S, Griffin, Danielle A, Herman, Adam C, Sahenk, Zarife, Rodino-Klapac, Louise R, Harper, Scott Q

“…RNAi emerged as a prospective molecular therapy nearly 15 years ago. Since then, two major RNAi platforms have been under development: oligonucleotides and…”
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Persistent Expression of FLAG-tagged Micro dystrophin in Nonhuman Primates Following Intramuscular and Vascular Delivery by Rodino-Klapac, Louise R, Montgomery, Chrystal L, Bremer, William G, Shontz, Kimberly M, Malik, Vinod, Davis, Nancy, Sprinkle, Spencer, Campbell, Katherine J, Sahenk, Zarife, Clark, K Reed, Walker, Christopher M, Mendell, Jerry R, Chicoine, Louis G

“…Animal models for Duchenne muscular dystrophy (DMD) have species limitations related to assessing function, immune response, and distribution of micro- or…”
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Impaired regeneration in calpain-3 null muscle is associated with perturbations in mTORC1 signaling and defective mitochondrial biogenesis by Yalvac, Mehmet E, Amornvit, Jakkrit, Braganza, Cilwyn, Chen, Lei, Hussain, Syed-Rehan A, Shontz, Kimberly M, Montgomery, Chrystal L, Flanigan, Kevin M, Lewis, Sarah, Sahenk, Zarife

“…Previous studies in patients with limb-girdle muscular dystrophy type 2A (LGMD2A) have suggested that calpain-3 (CAPN3) mutations result in aberrant…”
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Mutant HSPB1 overexpression in neurons is sufficient to cause age-related motor neuronopathy in mice by Srivastava, Amit K, Renusch, Samantha R, Naiman, Nicole E, Gu, Shuping, Sneh, Amita, Arnold, W. David, Sahenk, Zarife, Kolb, Stephen J

“…Abstract The small heat shock protein HSPB1 is a multifunctional, α-crystallin-based protein that has been shown to be neuroprotective in animal models of…”
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