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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
Mark Destabilizing Domains Enable Long-Term and Inert Regulation of GDNF Expression in the Brain
Quintino, Luis ^LU ; Namislo, Angrit ^LU ; Davidsson, Marcus ^LU ; Breger, Ludivine S. ^LU ; Kavanagh, Patrick ^LU ; Avallone, Martino ^LU ; Elgstrand-Wettergren, Erika ^LU ; Isaksson, Christina ^LU and Lundberg, Cecilia ^LU (2018) In Molecular Therapy - Methods and Clinical Development 11. p.29-39
- Contribution to journal › Article
Mark Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis : Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery
Cukras, Catherine ; Wiley, Henry E. ; Jeffrey, Brett G. ; Sen, H. Nida ; Turriff, Amy ; Zeng, Yong ; Vijayasarathy, Camasamudram ; Marangoni, Dario ; Ziccardi, Lucia and Kjellstrom, Sten ^LU , et al. (2018) In Molecular Therapy 26(9). p.2282-2294
- Contribution to journal › Article
Mark Development of Novel Therapies, Models, and Biomarkers for Osteoclast-Related Diseases
Löfvall, Henrik ^LU (2018) In Lund University, Faculty of Medicine Doctoral Dissertation Series 2018(108).
- Thesis › Doctoral thesis (compilation)
2017
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
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
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

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