Gene Therapy for Neurodegenerative Diseases: Slowing Down the Ticking Clock

por Martier, Raygene, Konstantinova, Pavlina
Publicado 2020

Hairpin-induced tRNA-mediated (HITME) recombination in HIV-1

por Konstantinova, Pavlina, de Haan, Peter, Das, Atze T., Berkhout, Ben
Publicado 2006

RNAi-mediated inhibition of HIV-1 by targeting partially complementary viral sequences

por Liu, Ying Poi, Gruber, Jens, Haasnoot, Joost, Konstantinova, Pavlina, Berkhout, Ben
Publicado 2009

Trans-inhibition of HIV-1 by a long hairpin RNA expressed within the viral genome

por Konstantinova, Pavlina, ter Brake, Olivier, Haasnoot, Joost, de Haan, Peter, Berkhout, Ben
Publicado 2007

Inhibition of HIV-1 by multiple siRNAs expressed from a single microRNA polycistron

por Liu, Ying Poi, Haasnoot, Joost, ter Brake, Olivier, Berkhout, Ben, Konstantinova, Pavlina
Publicado 2008

Optimization and comparison of knockdown efficacy between polymerase II expressed shRNA and artificial miRNA targeting luciferase and Apolipoprotein B100

por Maczuga, Piotr, Koornneef, Annemart, Borel, Florie, Petry, Harald, van Deventer, Sander, Ritsema, Tita, Konstantinova, Pavlina
Publicado 2012

In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver

por Liefhebber, Jolanda M., Martier, Raygene, Van der Zon, Tom, Keskin, Sonay, Huseinovic, Angelina, Lubelski, Jacek, Blits, Bas, Petry, Harald, Konstantinova, Pavlina
Publicado 2019

In vivo knock-down of multidrug resistance transporters ABCC1 and ABCC2 by AAV-delivered shRNAs and by artificial miRNAs

por Borel, Florie, van Logtenstein, Richard, Koornneef, Annemart, Maczuga, Piotr, Ritsema, Tita, Petry, Harald, van Deventer, Sander JH, Jansen, Peter LM, Konstantinova, Pavlina
Publicado 2011

AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models

por Spronck, Elisabeth A., Brouwers, Cynthia C., Vallès, Astrid, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.
Publicado 2019

AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes

por Keskin, Sonay, Brouwers, Cynthia C., Sogorb-Gonzalez, Marina, Martier, Raygene, Depla, Josse A., Vallès, Astrid, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.
Publicado 2019

PhP.B Enhanced Adeno-Associated Virus Mediated-Expression Following Systemic Delivery or Direct Brain Administration

por Pietersz, Kimberly L., Plessis, Francois Du, Pouw, Stephan M., Liefhebber, Jolanda M., van Deventer, Sander J., Martens, Gerard J. M., Konstantinova, Pavlina S., Blits, Bas
Publicado 2021

Rescue of spinal muscular atrophy mouse models with AAV9-Exon-specific U1 snRNA

por Donadon, Irving, Bussani, Erica, Riccardi, Federico, Licastro, Danilo, Romano, Giulia, Pianigiani, Giulia, Pinotti, Mirko, Konstantinova, Pavlina, Evers, Melvin, Lin, Shuo, Rüegg, Markus A, Pagani, Franco
Publicado 2019

Embedding siRNA sequences targeting Apolipoprotein B100 in shRNA and miRNA scaffolds results in differential processing and in vivo efficacy

por Maczuga, Piotr, Lubelski, Jacek, van Logtenstein, Richard, Borel, Florie, Blits, Bas, Fakkert, Erwin, Costessi, Adalberto, Butler, Derek, van Deventer, Sander, Petry, Harald, Koornneef, Annemart, Konstantinova, Pavlina
Publicado 2013

Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients

por Martier, Raygene, Liefhebber, Jolanda M., Miniarikova, Jana, van der Zon, Tom, Snapper, Jolanda, Kolder, Iris, Petry, Harald, van Deventer, Sander J., Evers, Melvin M., Konstantinova, Pavlina
Publicado 2019

Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy

por Martier, Raygene, Liefhebber, Jolanda M., García-Osta, Ana, Miniarikova, Jana, Cuadrado-Tejedor, Mar, Espelosin, Maria, Ursua, Susana, Petry, Harald, van Deventer, Sander J., Evers, Melvin M., Konstantinova, Pavlina
Publicado 2019

Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain

por Depla, Josse A., Sogorb-Gonzalez, Marina, Mulder, Lance A., Heine, Vivi M., Konstantinova, Pavlina, van Deventer, Sander J., Wolthers, Katja C., Pajkrt, Dasja, Sridhar, Adithya, Evers, Melvin M.
Publicado 2020

Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology

por Spronck, Elisabeth A., Vallès, Astrid, Lampen, Margit H., Montenegro-Miranda, Paula S., Keskin, Sonay, Heijink, Liesbeth, Evers, Melvin M., Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, de Haan, Martin
Publicado 2021

Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease

por Miniarikova, Jana, Zanella, Ilaria, Huseinovic, Angelina, van der Zon, Tom, Hanemaaijer, Evelyn, Martier, Raygene, Koornneef, Annemart, Southwell, Amber L, Hayden, Michael R, van Deventer, Sander J, Petry, Harald, Konstantinova, Pavlina
Publicado 2016

Secreted therapeutics: monitoring durability of microRNA-based gene therapies in the central nervous system

por Sogorb-Gonzalez, Marina, Vendrell-Tornero, Carlos, Snapper, Jolanda, Stam, Anouk, Keskin, Sonay, Miniarikova, Jana, Spronck, Elisabeth A, de Haan, Martin, Nieuwland, Rienk, Konstantinova, Pavlina, van Deventer1, Sander J, Evers, Melvin M, Vallès, Astrid
Publicado 2021

Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease

por Caron, Nicholas S, Southwell, Amber L, Brouwers, Cynthia C, Cengio, Louisa Dal, Xie, Yuanyun, Black, Hailey Findlay, Anderson, Lisa M, Ko, Seunghyun, Zhu, Xiang, van Deventer, Sander J, Evers, Melvin M, Konstantinova, Pavlina, Hayden, Michael R
Publicado 2020