Ronald J. Mandel

Long-term consequences of human alpha-synuclein overexpression in the primate ventral midbrain (2007)

Eslamboli, Andisheh, Romero-Ramos, Marina, Burger, Corinna, Bjorklund, Tomas, Muzyczka, Nicholas, Mandel, Ronald J., ...

Overexpression of human α-synuclein (α-syn) using recombinant adeno-associated viral (rAAV) vectors provides a novel tool to study neurodegenerative processes seen in Parkinson's disease and other...

Reversal of dyskinesias in an animal model of Parkinson's disease by continuous L-DOPA delivery using rAAV vectors (2005)

Carlsson, Thomas, Winkler, Christian, Burger, Corinna, Muzyczka, Nicholas, Mandel, Ronald J., Cenci, Angela, ...

Dyskinesias are a major complication of long-term l-3,4-dihydroxyphenylalanine (l-DOPA) treatment in Parkinson's disease, and are believed to result from the intermittent and pulsatile supply of...

Reversal of dyskinesias in an animal model of Parkinson's disease by continuous L-DOPA delivery using rAAV vectors (2005)

Carlsson, Thomas, Winkler, Christian, Burger, Corinna, Muzyczka, Nicholas, Mandel, Ronald J., Cenci, Angela, ...

Summary Dyskinesias are a major complication of long-term L-3,4-dihydroxyphenylalanine (L-DOPA) treatment in Parkinson's disease, and are believed to result from the intermittent and pulsatile supply...

Reversal of dyskinesias in an animal model of Parkinson's disease by continuous L-DOPA delivery using rAAV vectors (2005)

Carlsson, Thomas, Winkler, Christian, Burger, Corinna, Muzyczka, Nicholas, Mandel, Ronald J., Cenci, Angela, ...

Summary Dyskinesias are a major complication of long-term L-3,4-dihydroxyphenylalanine (L-DOPA) treatment in Parkinson's disease, and are believed to result from the intermittent and pulsatile supply...

Neuropathological and behavioral consequences of adeno-associated viral vector-mediated continuous intrastriatal neurotrophin delivery in a focal ischemia model in rats. (2002)

Andsberg, Gunnar, Kokaia, Zaal, Klein, Ronald L, Muzyczka, Nicholas, Lindvall, Olle, Mandel, Ronald J

Nerve growth factor (NGF) and brain-derived neurotrophic factor (BDNF) were continuously delivered to the striatum at biologically active levels via recombinant adeno-associated viral (rAAV) gene...

Parkinson-like neurodegeneration induced by targeted overexpression of alpha-synuclein in the nigrostriatal system. (2002)

Kirik, Deniz, Rosenblad, Carl, Burger, Corinna, Lundberg, Cecilia, Johansen, Teit E, Muzyczka, Nicholas, ...

Recombinant adeno-associated viral vectors display efficient tropism for transduction of the dopamine neurons of the substantia nigra. Taking advantage of this unique property of recombinant...

Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of L-dopa using rAAV-mediated gene transfer. (2002)

Kirik, Deniz, Georgievska, Biljana, Burger, Corinna, Winkler, Christian, Muzyczka, Nicholas, Mandel, Ronald J, ...

Intrastriatal delivery of the tyrosine hydroxylase gene by viral vectors is being explored as a tool for local delivery of L-dopa in animals with lesions of the nigrostriatal pathway. The functional...

Self-Inactivating Lentivirus Vector for Safe and Efficient In Vivo Gene Delivery

Zufferey, Romain, Dull, Thomas, Mandel, Ronald J., Bukovsky, Anatoly, Quiroz, Dulce, Naldini, Luigi, ...

In vivo transduction of nondividing cells by human immunodeficiency virus type 1 (HIV-1)-based vectors results in transgene expression that is stable over several months. However, the use of HIV-1...

Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of l-dopa using rAAV-mediated gene transfer

Kirik, Deniz, Georgievska, Biljana, Burger, Corinna, Winkler, Christian, Muzyczka, Nicholas, Mandel, Ronald J., ...

Intrastriatal delivery of the tyrosine hydroxylase gene by viral vectors is being explored as a tool for local delivery of l-dopa in animals with lesions of the nigrostriatal pathway. The functional...

Nigrostriatal α-synucleinopathy induced by viral vector-mediated overexpression of human α-synuclein: A new primate model of Parkinson's disease

Kirik, Deniz, Annett, Lucy E., Burger, Corinna, Muzyczka, Nicholas, Mandel, Ronald J., Björklund, Anders

We used a high-titer recombinant adeno-associated virus (rAAV) vector to express WT or mutant human α-synuclein in the substantia nigra of adult marmosets. The α-synuclein protein was expressed in...

Circulating Anti-Wild-Type Adeno-Associated Virus Type 2 (AAV2) Antibodies Inhibit Recombinant AAV2 (rAAV2)-Mediated, but Not rAAV5-Mediated, Gene Transfer in the Brain

Peden, Carmen S., Burger, Corinna, Muzyczka, Nicholas, Mandel, Ronald J.

Epidemiological studies report that 80% of the population maintains antibodies (Ab) to wild-type (wt) adeno-associated virus type 2 (AAV2), with 30% expressing neutralizing Ab (NAb). The blood-brain...

Self-Inactivating Lentivirus Vector for Safe and Efficient In Vivo Gene Delivery

Zufferey, Romain, Dull, Thomas, Mandel, Ronald J., Bukovsky, Anatoly, Quiroz, Dulce, Naldini, Luigi, ...

In vivo transduction of nondividing cells by human immunodeficiency virus type 1 (HIV-1)-based vectors results in transgene expression that is stable over several months. However, the use of HIV-1...

Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of l-dopa using rAAV-mediated gene transfer

Kirik, Deniz, Georgievska, Biljana, Burger, Corinna, Winkler, Christian, Muzyczka, Nicholas, Mandel, Ronald J., ...

Intrastriatal delivery of the tyrosine hydroxylase gene by viral vectors is being explored as a tool for local delivery of l-dopa in animals with lesions of the nigrostriatal pathway. The functional...

Nigrostriatal α-synucleinopathy induced by viral vector-mediated overexpression of human α-synuclein: A new primate model of Parkinson's disease

Kirik, Deniz, Annett, Lucy E., Burger, Corinna, Muzyczka, Nicholas, Mandel, Ronald J., Björklund, Anders

We used a high-titer recombinant adeno-associated virus (rAAV) vector to express WT or mutant human α-synuclein in the substantia nigra of adult marmosets. The α-synuclein protein was expressed in...

Circulating Anti-Wild-Type Adeno-Associated Virus Type 2 (AAV2) Antibodies Inhibit Recombinant AAV2 (rAAV2)-Mediated, but Not rAAV5-Mediated, Gene Transfer in the Brain

Peden, Carmen S., Burger, Corinna, Muzyczka, Nicholas, Mandel, Ronald J.

Epidemiological studies report that 80% of the population maintains antibodies (Ab) to wild-type (wt) adeno-associated virus type 2 (AAV2), with 30% expressing neutralizing Ab (NAb). The blood-brain...

The phosphorylation state of Ser-129 in human α-synuclein determines neurodegeneration in a rat model of Parkinson disease

Gorbatyuk, Oleg S., Li, Shoudong, Sullivan, Layla F., Chen, Weijun, Kondrikova, Galina, Manfredsson, Fredric P., ...

Studies have shown that α-synuclein (α-syn) deposited in Lewy bodies in brain tissue from patients with Parkinson disease (PD) is extensively phosphorylated at Ser-129. We used recombinant...