Search Results - "Castellanos Rivera, Ruth M."

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy by Nelson, Christopher E., Wu, Yaoying, Gemberling, Matthew P., Oliver, Matthew L., Waller, Matthew A., Bohning, Joel D., Robinson-Hamm, Jacqueline N., Bulaklak, Karen, Castellanos Rivera, Ruth M., Collier, Joel H., Asokan, Aravind, Gersbach, Charles A.

“…Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for therapeutic genome editing. There have been several recent reports of genome…”
Get full text

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy by Nelson, Christopher E., Hakim, Chady H., Ousterout, David G., Thakore, Pratiksha I., Moreb, Eirik A., Rivera, Ruth M. Castellanos, Madhavan, Sarina, Pan, Xiufang, Ran, F. Ann, Yan, Winston X., Asokan, Aravind, Zhang, Feng, Duan, Dongsheng, Gersbach, Charles A.

“…Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000 male births and caused by mutations in the dystrophin gene. Genome…”
Get full text

Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion by Tse, Longping Victor, Klinc, Kelli A., Madigan, Victoria J., Rivera, Ruth M. Castellanos, Wells, Lindsey F., Havlik, L. Patrick, Smith, J. Kennon, Agbandje-McKenna, Mavis, Asokan, Aravind

“…Preexisting neutralizing antibodies (NAbs) against adeno-associated viruses (AAVs) pose a major, unresolved challenge that restricts patient enrollment in gene…”
Get full text

Aquaporin-4-dependent glymphatic solute transport in the rodent brain by Mestre, Humberto, Hablitz, Lauren M, Xavier, Anna Lr, Feng, Weixi, Zou, Wenyan, Pu, Tinglin, Monai, Hiromu, Murlidharan, Giridhar, Castellanos Rivera, Ruth M, Simon, Matthew J, Pike, Martin M, Plá, Virginia, Du, Ting, Kress, Benjamin T, Wang, Xiaowen, Plog, Benjamin A, Thrane, Alexander S, Lundgaard, Iben, Abe, Yoichiro, Yasui, Masato, Thomas, John H, Xiao, Ming, Hirase, Hajime, Asokan, Aravind, Iliff, Jeffrey J, Nedergaard, Maiken

“…The glymphatic system is a brain-wide clearance pathway; its impairment contributes to the accumulation of amyloid-β. Influx of cerebrospinal fluid (CSF)…”
Get full text

Tissue-Dependent Expression and Translation of Circular RNAs with Recombinant AAV Vectors In Vivo by Meganck, Rita M., Borchardt, Erin K., Castellanos Rivera, Ruth M., Scalabrino, Miranda L., Wilusz, Jeremy E., Marzluff, William F., Asokan, Aravind

“…Circular RNAs (circRNAs) are long-lived, covalently closed RNAs that are abundantly expressed and evolutionarily conserved across eukaryotes. Possible…”
Get full text

Transcriptional regulator RBP-J regulates the number and plasticity of renin cells by Castellanos Rivera, Ruth M, Monteagudo, Maria C, Pentz, Ellen S, Glenn, Sean T, Gross, Kenneth W, Carretero, Oscar, Sequeira-Lopez, Maria Luisa S, Gomez, R Ariel

“…Renin-expressing cells are crucial in the control of blood pressure and fluid-electrolyte homeostasis. Notch receptors convey cell-cell signals that may…”
Get full text

Structure-guided AAV capsid evolution strategies for enhanced CNS gene delivery by Gonzalez, Trevor J., Mitchell-Dick, Aaron, Blondel, Leo O., Fanous, Marco M., Hull, Joshua A., Oh, Daniel K., Moller-Tank, Sven, Castellanos Rivera, Ruth M., Piedrahita, Jorge A., Asokan, Aravind

“…Over the past 5 years, our laboratory has systematically developed a structure-guided library approach to evolve new adeno-associated virus (AAV) capsids with…”
Get full text

Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing by Gonzalez, Trevor J., Simon, Katherine E., Blondel, Leo O., Fanous, Marco M., Roger, Angela L., Maysonet, Maribel Santiago, Devlin, Garth W., Smith, Timothy J., Oh, Daniel K., Havlik, L. Patrick, Castellanos Rivera, Ruth M., Piedrahita, Jorge A., ElMallah, Mai K., Gersbach, Charles A., Asokan, Aravind

“…Recombinant adeno-associated viral (AAV) vectors are a promising gene delivery platform, but ongoing clinical trials continue to highlight a relatively narrow…”
Get full text

Functional Analysis of the Putative Integrin Recognition Motif on Adeno-associated Virus 9 by Shen, Shen, Berry, Garrett E., Castellanos Rivera, Ruth M., Cheung, Roland Y., Troupes, Andrew N., Brown, Sarah M., Kafri, Tal, Asokan, Aravind

“…Adeno-associated viruses (AAVs) display a highly conserved NGR motif on the capsid surface. Earlier studies have established this tripeptide motif as being…”
Get full text

Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier by Albright, Blake H., Storey, Claire M., Murlidharan, Giridhar, Castellanos Rivera, Ruth M., Berry, Garrett E., Madigan, Victoria J., Asokan, Aravind

“…Effective gene delivery to the CNS by intravenously administered adeno-associated virus (AAV) vectors requires crossing the blood-brain barrier (BBB). To…”
Get full text

Recombination signal binding protein for Ig-κJ region regulates juxtaglomerular cell phenotype by activating the myo-endocrine program and suppressing ectopic gene expression by Castellanos-Rivera, Ruth M, Pentz, Ellen S, Lin, Eugene, Gross, Kenneth W, Medrano, Silvia, Yu, Jing, Sequeira-Lopez, Maria Luisa S, Gomez, R Ariel

“…Recombination signal binding protein for Ig-κJ region (RBP-J), the major downstream effector of Notch signaling, is necessary to maintain the number of…”
Get full text

194. Lymphatic Transport Mediates Systemic Leakage of Peripherally Administered AAV9 Vectors by Castellanos Rivera, Ruth M., Rao, Lavanya, Murlidharan, Giridhar, Corriher, Travis, Asokan, Aravind

Get full text

504. Restoration of Dystrophin Expression by Gene Editing with S. aureus Cas9 in Models of Duchenne Muscular Dystrophy by Robinson-Hamm, Jacqueline N., Nelson, Christopher E., Rivera, Ruth M. Castellanos, Aartsma-Rus, Annemieke, Asokan, Aravind, Gersbach, Charles A.

Get full text

Abstract 38: Rbp-j Regulates the Genetic Program of the Myo-epithelioid JG Cell by Castellanos Rivera, Ruth M, Pentz, Ellen S., Gross, Kenneth W., Medrano, Silvia, Yu, Jing, Sequeira-Lopez, Maria Luisa S., Gomez, R. Ariel

“…Abstract only RBP-J , the major downstream effector of Notch signaling, is necessary to maintain the number of juxtaglomerular (JG) cells. In addition, RBP-J…”
Get full text

RBP-J regulates the identity and plasticity of renin cells: A fundamental mechanism to control homeostasis by Castellanos Rivera, Ruth M

“…Renin is a hormone/enzyme crucial for maintaining blood pressure and fluid electrolyte homeostasis. In adult animals, renin-expressing cells are restricted to…”
Get full text