367. A Dopamine Gene Therapy for Advanced PD: 4 Years Phase I/II Clinical Update von Palfi, Stéphane, Gurruchaga, Jean Marc, Ralph, Scott Gs, Watts, Colin, Buttery, Philip, Lepetit, Helene, Miskin, James, Gouello, Gaetane, Lavisse, Sonia, Fenelon, Gilles, Thiriez, Claire, Brugières, Pierre, Barker, Roger, Hantraye, Philippe, Mitrophanous, Kyriacos

Volltext

VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model von Azzouz, Mimoun, Ralph, G. Scott, Storkebaum, Erik, Walmsley, Lucy E., Mitrophanous, Kyriacos A., Kingsman, Susan M., Carmeliet, Peter, Mazarakis, Nicholas D.

Volltext

Removal of AMPA Receptors (AMPARs) from Synapses Is Preceded by Transient Endocytosis of Extrasynaptic AMPARs von Ashby, Michael C, De La Rue, Sarah A, Ralph, G. Scott, Uney, James, Collingridge, Graham L, Henley, Jeremy M

Volltext

Model organisms contribute to diagnosis and discovery in the undiagnosed diseases network: current state and a future vision von Baldridge, Dustin, Wangler, Michael F, Bowman, Angela N, Yamamoto, Shinya, Schedl, Tim, Pak, Stephen C, Postlethwait, John H, Shin, Jimann, Solnica-Krezel, Lilianna, Bellen, Hugo J, Westerfield, Monte

Volltext

Bi-allelic variants in INTS11 are associated with a complex neurological disorder von Tepe, Burak, Cogne, Benjamin, Neil, Jennifer E., Walsh, Christopher A., Magal, Nurit, Drasinover, Valerie, Schwab, Tanya, Schmitz, Chris, Clark, Karl, Blanc, Pierre, Bademci, Guney, Balasubramanyam, Ashok, Baldridge, Dustin, Bayrak-Toydemir, Pinar, Beck, Anita, Behrens, Edward, Bican, Anna, Bivona, Stephanie, Blue, Elizabeth, Boyd, Brenna, Briere, Lauren C., Brown, Gabrielle, Byers, Peter, Byrd, William E., Carey, John, Clark, Gary D., Coakley, Terra R., Colley, Heather A., Craigen, William J., Cunningham, Michael, Dasari, Surendra, Dayal, Jyoti G., Deardorff, Matthew, Dipple, Katrina, Doherty, Daniel, Doss, Argenia L., Emrick, Lisa T., Fernandez, Liliana, Forghani, Irman, Glass, Ian, Gochuico, Bernadette, Golden-Grant, Katie, Goldrich, Madison P., Gutierrez, Irma, Hamid, Rizwan, Hayes, Nichole, Hom, Jason, Horike-Pyne, Martha, Isasi, Rosario, Jarvik, Jeffrey, Jobanputra, Vaidehi, Karaviti, Lefkothea, Kiley, Dana, Kobren, Shilpa N., Krasnewich, Donna M., LeBlanc, Kimberly, Levitt, Roy, Mahoney, Rachel, Malicdan, May Christine V., Mamounas, Laura A., Mao, Rong, Maravilla, Kenneth, Marom, Ronit, Martin, Martin G., Martínez-Agosto, Julian A., Merritt, J. Lawrence, Morava, Eva, Newman, John H., Nickerson, Deborah, Novacic, Donna, Oglesbee, Devin, Pace, Laura, Palmer, Christina GS, Papp, Jeanette C., Rao, Deepak A., Renteria, Genecee, Reuter, Chloe M., Robertson, Amy K., Rosenfeld, Jill A., Rossignol, Francis, Ruzhnikov, Maura, Scott, C. Ron, Shin, Jimann, Sinsheimer, Janet S., Sullivan, Jennifer A., Sun, Angela, Tabor, Holly K., Telischi, Fred, Toro, Camilo, Tucker, Brianna M., Urv, Tiina K., Vogel, Tiphanie P., Walker, Melissa, Wangler, Michael F., Perry, Katherine Wesseling, Westerfield, Monte, Wolfe, Lynne A., Worley, Kim, Xiao, Changrui, Zuchner, Stephan

Volltext

Heterozygous variants in MYBPC1 are associated with an expanded neuromuscular phenotype beyond arthrogryposis von Schoch, Kelly, Sullivan, Jennifer, Kranz, Peter, Kontrogianni‐Konstantopoulos, Aikaterini, Adams, David R., Allard, Patrick, Balasubramanyam, Ashok, Barbouth, Deborah, Bellen, Hugo J., Bernstein, Jonathan A., Bick, David P., Bonner, Devon, Boone, Braden E., Brown, Donna M., Brush, Matthew, Burke, Elizabeth A., Butte, Manish J., Chang, Ta Chen Peter, Clark, Gary D., Cobban, Laurel A., Cogan, Joy D., Cope, Heidi, Craigen, William J., Dasari, Surendra, Davids, Mariska, Dhar, Shweta U., Dorrani, Naghmeh, Draper, David D., Dries, Annika M., Duncan, Laura, Emrick, Lisa T., Eng, Christine M., Esteves, Cecilia, Estwick, Tyra, Fernandez, Liliana, Fieg, Elizabeth L., Forghani, Irman, Godfrey, Rena A., Gourdine, Jean‐Philippe F., Gropman, Andrea L., Haendel, Melissa, Hanchard, Neil A., High, Frances, Hom, Jason, Huang, Alden, Isasi, Rosario, Kelley, Emily G., Lanpher, Brendan C., Lanza, Ian R., Lau, C. Christopher, LeBlanc, Kimberly, Lee, Hane, Levitt, Roy, Lewis, Richard A., Liu, Pengfei, Maas, Richard L., Macnamara, Ellen F., Majcherska, Marta M., Malicdan, May Christine V., Manolio, Teri A., Markello, Thomas C., Marom, Ronit, Martínez‐Agosto, Julian A., May, Thomas, McCauley, Jacob, McConkie‐Rosell, Allyn, McCormack, Colleen E., Might, Matthew, Murdock, David R., Nath, Avi, Nelson, Stan F., Newberry, J. Scott, Pak, Stephen, Pallais, J. Carl, Parker, Neil H., Potocki, Lorraine, Pusey, Barbara N., Renteria, Genecee, Robertson, Amy K., Sacco, Ralph, Sampson, Jacinda B., Schaechter, Judy, Schedl, Timothy, Silverman, Edwin K., Stoler, Joan M., Sullivan, Jennifer A., Sweetser, David A., Tekin, Mustafa, Thorson, Willa, Toro, Camilo, Tran, Alyssa A., Urv, Tiina K., Walley, Nicole M., Walker, Melissa, Waters, Katrina M., Wegner, Daniel, Wheeler, Matthew T., Yamamoto, Shinya, Yang, John, Yoon, Amanda J.

Volltext

EFEMP1 haploinsufficiency causes a Marfan‐like hereditary connective tissue disorder von Forghani, Irman, Adam, Margaret, Azamian, Mahshid S., Baker, Eva, Baldridge, Dustin, Bale, Jim, Beck, Anita, Berg‐Rood, Beverly, Berry, Gerard T., Bican, Anna, Boyd, Brenna, Briere, Lauren C., Burke, Elizabeth A., Burrage, Lindsay C., Byers, Peter, Carey, John, Chang, Ta Chen Peter, Cogan, Joy D., Colley, Heather A., D'Souza, Precilla, Dasari, Surendra, Dell'Angelica, Esteban C., Duncan, Laura, Eckstein, David J., Emrick, Lisa T., Eng, Christine M., Esteves, Cecilia, Fernandez, Liliana, Fieg, Elizabeth L., Forghani, Irman, Gochuico, Bernadette, Grajewski, Alana, Hahn, Sihoun, Hamid, Rizwan, High, Frances, Hing, Anne, Hisama, Fuki M., Holm, Ingrid A., Huang, Yong, Jamal, Fariha, Jayadev, Suman, Kohler, Jennefer N., Krakow, Deborah, Kravets, Elijah, Koziura, Mary, Lam, Byron, Lam, Christina, Lanza, Ian R., LeBlanc, Kimberly, Lee, Brendan H., Lee, Hane, Levitt, Roy, Liu, Pengfei, Maas, Richard L., Macnamara, Ellen F., Maduro, Valerie V., Majcherska, Marta M., Manolio, Teri A., Marth, Gabor, Martínez‐Agosto, Julian A., Marwaha, Shruti, McCauley, Jacob, McConkie‐Rosell, Allyn, McGee, Elisabeth, Might, Matthew, Mirzaa, Ghayda, Moretti, Paolo M., Mosbrook‐Davis, Deborah, Murdock, David R., Nieves‐Rodriguez, Shirley, Parker, Neil H., Phillips, John A., Power, Bradley, Pusey, Barbara N., Quinlan, Aaron, Raskind, Wendy, Sacco, Ralph, Sampson, Jacinda B., Saporta, Mario, Shin, Jimann, Silverman, Edwin K., Sinsheimer, Janet S., Solem, Emily, Stoler, Joan M., Sun, Angela, Tabor, Holly K., Tan, Amelia L. M., Telischi, Fred, Tifft, Cynthia J., Toro, Camilo, Tucker, Brianna M., Vogel, Tiphanie P., Wambach, Jennifer, Wang, Lee‐kai, Wegner, Daniel, Wenger, Tara, Westerfield, Monte, Wolfe, Lynne A., Zein, Wadih, Zuchner, Stephan

Volltext

Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy von Azzouz, Mimoun, Le, Thanh, Ralph, G. Scott, Walmsley, Lucy, Monani, Umrao R., Lee, Debbie C.P., Wilkes, Fraser, Mitrophanous, Kyriacos A., Kingsman, Susan M., Burghes, Arthur H.M., Mazarakis, Nicholas D.

Volltext

Surface expression of AMPA receptors in hippocampal neurons is regulated by an NSF-dependent mechanism von Noel, J, Ralph, G S, Pickard, L, Williams, J, Molnar, E, Uney, J B, Collingridge, G L, Henley, J M

Volltext

Germline and Somatic Mutations of the STK11/LKB1 Peutz-Jeghers Gene in Pancreatic and Biliary Cancers von Su, Gloria H., Hruban, Ralph H., Bansal, Ravi K., Bova, G. Steven, Tang, David J., Shekher, Manu C., Westerman, Anne Marie, Entius, Mark M., Goggins, Michael, Yeo, Charles J., Kern, Scott E.

Volltext

Tissue inhibitor of metalloproteinase 1 inhibits excitotoxic cell death in neurons von Tan, Hiang Khoon, Heywood, Darren, Ralph, G.Scott, Bienemann, Alison, Baker, Andrew H, Uney, James B

Volltext

Lentiviral-Mediated Delivery of Bcl-2 or GDNF Protects against Excitotoxicity in the Rat Hippocampus von Wong, Liang-Fong, Scott Ralph, G., Walmsley, Lucy E., Bienemann, Alison S., Parham, Stephen, Kingsman, Susan M., Uney, James B., Mazarakis, Nicholas D.

Volltext

DPC4 gene in various tumor types von SCHUTTE, M, HRUBAN, R. H, SIDRANSKY, D, CASERO, R. A, MELTZER, P. S, HAHN, S. A, KERN, S. E, HEDRICK, L, CHO, K. R, GYONGYI MOLNAR NADASDY, WEINSTEIN, C. L, BOVA, G. S, ISAACS, W. B, CAIRNS, P, NAWROZ, H

Volltext

Infective Endocarditis Due to Staphylococcus aureus: 59 Prospectively Identified Cases with Follow-up von Fowler, Vance G., Sanders, Linda L., Kong, Li Kuo, McClelland, R. Scott, Gottlieb, Geoffrey S., Li, Jennifer, Ryan, Thomas, Sexton, Daniel J., Roussakis, Georges, Harrell, Lizzie J., Corey, G. Ralph

Volltext

Therapeutic gene silencing in neurological disorders, using interfering RNA von RALPH, G. Scott, MAZARAKIS, Nicholas D, AZZOUZ, Mimoun

Volltext

Recurrent Staphylococcus aureus Bacteremia: Pulsed-Field Gel Electrophoresis Findings in 29 Patients von Fowler, Vance G., Li Kuo Kong, Corey, G. Ralph, Gottlieb, Geoffrey S., McClelland, R. Scott, Sexton, Daniel J., Gesty-Palmer, Diane, Harrell, Lizzie J.

Volltext

Hyperhydration: tolerance and cardiovascular effects during uncompensable exercise-heat stress von Latzka, William A, Sawka, Michael N, Montain, Scott J, Skrinar, Gary S, Fielding, Roger A, Matott, Ralph P, Pandolf, Kent B

Volltext

Targeting of tetracycline-regulatable transgene expression specifically to neuronal and glial cell populations using adenoviral vectors von Ralph, G Scott, Bienemann, Alison, Harding, Tom C, Hopton, Maggie, Henley, Jeremy, Uney, James B

Volltext

Prospective Analysis of Staphylococcus aureus Bacteremia in Nonneutropenic Adults With Malignancy von GOPAL, A. K, FOWLER, V. G, SEXTON, D. J, COREY, G. R, SHAH, M, GESTY-PALMER, D, MARR, K. A, MCCLELLAND, R. S, LI KUO KONG, GOTTLIEB, G. S, LANCLOS, K, LI, J

Volltext

Hyperhydration: thermoregulatory effects during compensable exercise-heat stress von Latzka, William A, Sawka, Michael N, Montain, Scott J, Skrinar, Gary S, Fielding, Roger A, Matott, Ralph P, Pandolf, Kent B

Volltext