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2018
Mark Haemophilia B : Where are we now and what does the future hold?
Dolan, Gerry ; Benson, Gary ; Duffy, Anne ; Hermans, Cedric ; Jiménez-Yuste, Victor ; Lambert, Thierry ; Ljung, Rolf ^LU ; Morfini, Massimo and Zupančić Šalek, Silva (2018) In Blood Reviews 32(1). p.52-60
- Contribution to journal › Scientific review
2017
Mark Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia
Debnath, Shubhranshu ^LU ; Jaako, Pekka ^LU ; Siva, Kavitha ^LU ; Rothe, Michael ; Chen, Jun ^LU ; Dahl, Maria ^LU ; Gaspar, H. Bobby ; Flygare, Johan ^LU ; Schambach, Axel and Karlsson, Stefan ^LU (2017) In Molecular Therapy 25(8). p.1805-1814
- Contribution to journal › Article
Mark Rearing light intensity affects inner retinal pathology in a mouse model of x-linked retinoschisis but does not alter gene therapy outcome
Marangoni, Dario ; Yong, Zeng ; Kjellström, Sten ^LU ; Vijayasarathy, Camasamudram ; Sieving, Paul A. and Bush, Ronald A (2017) In Investigative Ophthalmology and Visual Science 58(3). p.1656-1664
- Contribution to journal › Article
Mark The clinical phenotype of CNGA3-related achromatopsia : Pretreatment characterization in preparation of a gene replacement therapy trial
Zobor, Ditta ; Werner, Annette ; Stanzial, Franco ; Benedicenti, Francesco ; Rudolph, Günther ; Kellner, Ulrich ; Hamel, Christian P ; Andréasson, Sten ^LU ; Zobor, Gergely and Strasser, Torsten , et al. (2017) In Investigative Ophthalmology and Visual Science 58(2). p.821-832
- Contribution to journal › Article
2016
Mark Regulation and Function of Lentiviral Vector-Mediated TCIRG1 Expression in Osteoclasts from Patients with Infantile Malignant Osteopetrosis : Implications for Gene Therapy
Thudium, Christian Schneider ^LU ; Moscatelli, Ilana ^LU ; Löfvall, Henrik ^LU ; Kertész, Zsuzsanna ^LU ; Montano, Carmen ^LU ; Bjurström, Carmen Flores ^LU ; Karsdal, Morten Asser ; Schulz, Ansgar ; Richter, Johan ^LU and Henriksen, Kim (2016) In Calcified Tissue International 99(6). p.638-648
- Contribution to journal › Article
2015
Mark Cell and gene therapy for blood disorders. Modeling of novel treatment strategies for Gaucher type 1 disease and Diamond-Blackfan anemia in mice.
Dahl, Maria ^LU (2015) In Lund University Faculty of Medicine Doctoral Dissertation Series 2015:92.
- Thesis › Doctoral thesis (compilation)
Mark Non-Viral Deoxyribonucleoside Kinases - Diversity and Practical Use
Slot Christiansen, Louise ^LU ; Munch-Petersen, Birgitte ^LU and Knecht, Wolfgang ^LU (2015) In Journal of Genetics and Genomics 42(5). p.235-248
- Contribution to journal › Scientific review
2014
Mark Development of Novel Models for Studying Osteoclasts
Thudium, Christian ^LU (2014) In Lund University Faculty of Medicine Doctoral Dissertation Series 2014:111.
- Thesis › Doctoral thesis (compilation)
Mark Factor VIII therapy for hemophilia A: current and future issues
Aledort, Louis ; Ljung, Rolf ^LU ; Mann, Kenneth and Pipe, Steven (2014) In Expert Review of Hematology 7(3). p.373-385
- Contribution to journal › Scientific review
2013
Mark Neuropeptides and neurotrophic factors in epilepsy: seizure suppressant actions of VEGF, NPY and galanin
Nikitidou, Litsa ^LU (2013) In Lund University Faculty of Medicine Doctoral Dissertation Series 2013:33.
- Thesis › Doctoral thesis (compilation)

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