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- 2018
-
Mark
Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis : Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery
(
- Contribution to journal › Article
-
Mark
Destabilizing Domains Enable Long-Term and Inert Regulation of GDNF Expression in the Brain
(
- Contribution to journal › Article
-
Mark
Development of Novel Therapies, Models, and Biomarkers for Osteoclast-Related Diseases
(
- 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
(
- Contribution to journal › Article
-
Mark
The clinical phenotype of CNGA3-related achromatopsia : Pretreatment characterization in preparation of a gene replacement therapy trial
(
- 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
(
- 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
(
- 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.
(
- Thesis › Doctoral thesis (compilation)
-
Mark
Non-Viral Deoxyribonucleoside Kinases - Diversity and Practical Use
(
- Contribution to journal › Scientific review
- 2014
-
Mark
Development of Novel Models for Studying Osteoclasts
(
- Thesis › Doctoral thesis (compilation)
-
Mark
Factor VIII therapy for hemophilia A: current and future issues
(
- Contribution to journal › Scientific review
- 2013
-
Mark
Neuropeptides and neurotrophic factors in epilepsy: seizure suppressant actions of VEGF, NPY and galanin
(
- Thesis › Doctoral thesis (compilation)
- 2012
-
Mark
Targeting the hematopoietic stem cell to correct osteopetrosis
(
- Thesis › Doctoral thesis (compilation)
-
Mark
Combined gene overexpression of neuropeptide Y and its receptor Y5 in the hippocampus suppresses seizures
(
- Contribution to journal › Article
-
Mark
How can measurement, monitoring, modeling and control advance cell culture in industrial biotechnology?
(
- Contribution to journal › Article
-
Mark
Novel disease-specific promoters for use in gene therapy for Parkinson's disease.
(
- Contribution to journal › Article
- 2011
-
Mark
Stem cell self-renewal: lessons from bone marrow, gut and iPS toward clinical applications
(
- Contribution to journal › Scientific review
-
Mark
Inclusion of a single-tail amino acid-based amphiphile in a lipoplex formulation: Effects on transfection efficiency and physicochemical properties
(
- Contribution to journal › Article
- 2010
-
Mark
Gene therapy in a new model of delayed wound healing
(
- Thesis › Doctoral thesis (compilation)
-
Mark
X-LINKED RETINOSCHISIS ELECTROPHYSIOLOGY, MOLECULAR GENETICS AND TREATMENT
(
- Thesis › Doctoral thesis (compilation)