Curative gene therapies for rare diseases

Autor Maldonado, Rocio, Jalil, Sami, Wartiovaara, Kirmo
Vydáno v J Community Genet (2020)

PlasmaDNA: a free, cross-platform plasmid manipulation program for molecular biology laboratories

Autor Angers-Loustau, Alexandre, Rainy, Jeffrey, Wartiovaara, Kirmo
Vydáno 2007

Timing of the Cell Cycle Exit of Differentiating Hippocampal Neural Stem Cells

Autor Järvinen, Elina, Angers-Loustau, Alexandre, Osiceanu, Ana-Maria, Wartiovaara, Kirmo
Vydáno 2010

N-myc Promotes Survival and Induces S-Phase Entry of Postmitotic Sympathetic Neurons

Autor Wartiovaara, Kirmo, Barnabé-Heider, Fanie, Miller, Freda D., Kaplan, David R.
Vydáno v J Neurosci (2002)

Conditionally Stabilized dCas9 Activator for Controlling Gene Expression in Human Cell Reprogramming and Differentiation

Autor Balboa, Diego, Weltner, Jere, Eurola, Solja, Trokovic, Ras, Wartiovaara, Kirmo, Otonkoski, Timo
Vydáno v Stem Cell Reports (2015)

Moving towards a cure in genetics: what is needed to bring somatic gene therapy to the clinic?

Autor Cornel, Martina C., Howard, Heidi C., Lim, Daniel, Bonham, Vence L., Wartiovaara, Kirmo
Vydáno v Eur J Hum Genet (2018)

Unraveling the disease pathogenesis behind lethal hydrolethalus syndrome revealed multiple changes in molecular and cellular level

Autor Honkala, Heli, Lahtela, Jenni, Fox, Heli, Gentile, Massimiliano, Pakkasjärvi, Niklas, Salonen, Riitta, Wartiovaara, Kirmo, Jauhiainen, Matti, Kestilä, Marjo
Vydáno 2009

Insulin mutations impair beta-cell development in a patient-derived iPSC model of neonatal diabetes

Autor Balboa, Diego, Saarimäki-Vire, Jonna, Borshagovski, Daniel, Survila, Mantas, Lindholm, Päivi, Galli, Emilia, Eurola, Solja, Ustinov, Jarkko, Grym, Heli, Huopio, Hanna, Partanen, Juha, Wartiovaara, Kirmo, Otonkoski, Timo
Vydáno v eLife (2018)

CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells

Autor Chung, Jennifer E., Magis, Wendy, Vu, Jonathan, Heo, Seok-Jin, Wartiovaara, Kirmo, Walters, Mark C., Kurita, Ryo, Nakamura, Yukio, Boffelli, Dario, Martin, David I. K., Corn, Jacob E., DeWitt, Mark A.
Vydáno v PLoS One (2019)