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Blood
|
December 3, 2011
AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage
Christopher Binny, Jenny McIntosh, Marco Della Peruta, et al.
Gene Therapy
|
January 11, 2023
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
Jey M Jeyakumar, Azadeh Kia, Lawrence C S Tam, et al.
Gene Therapy
|
July 20, 2018
The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins
Michael C Paul-Smith, Kamila M Pytel, Jean-François Gelinas, et al.
Science Translational Medicine
|
August 9, 2023
Adeno-associated virus gene therapy prevents progression of kidney disease in genetic models of nephrotic syndrome
Wen Y Ding, Valeryia Kuzmuk, Sarah Hunter, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 2, 2011
Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques
Citra N Z Mattar, Amit C Nathwani, Simon N Waddington, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 20, 2011
Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins
Amit C Nathwani, Cecilia Rosales, Jenny McIntosh, et al.
Blood
|
February 22, 2013
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
Jenny McIntosh, Peter J Lenting, Cecilia Rosales, et al.
The New England Journal of Medicine
|
June 11, 2025
Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B
Ulrike M Reiss, Andrew M Davidoff, Edward G D Tuddenham, et al.
The New England Journal of Medicine
|
November 20, 2014
Long-term safety and efficacy of factor IX gene therapy in hemophilia B
Amit C Nathwani, Ulreke M Reiss, Edward G D Tuddenham, et al.
The New England Journal of Medicine
|
December 14, 2011
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
Amit C Nathwani, Edward G D Tuddenham, Savita Rangarajan, et al.
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of 3
Search research articles
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Showing results (21-30 of 30) with videos related to
Sort By:
Page
of 3
You have reached the last page of results.
This site can display upto 30 results.
Blood
|
December 3, 2011
AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage
Christopher Binny, Jenny McIntosh, Marco Della Peruta, et al.
Gene Therapy
|
January 11, 2023
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
Jey M Jeyakumar, Azadeh Kia, Lawrence C S Tam, et al.
Gene Therapy
|
July 20, 2018
The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins
Michael C Paul-Smith, Kamila M Pytel, Jean-François Gelinas, et al.
Science Translational Medicine
|
August 9, 2023
Adeno-associated virus gene therapy prevents progression of kidney disease in genetic models of nephrotic syndrome
Wen Y Ding, Valeryia Kuzmuk, Sarah Hunter, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 2, 2011
Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques
Citra N Z Mattar, Amit C Nathwani, Simon N Waddington, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 20, 2011
Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins
Amit C Nathwani, Cecilia Rosales, Jenny McIntosh, et al.
Blood
|
February 22, 2013
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
Jenny McIntosh, Peter J Lenting, Cecilia Rosales, et al.
The New England Journal of Medicine
|
June 11, 2025
Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B
Ulrike M Reiss, Andrew M Davidoff, Edward G D Tuddenham, et al.
The New England Journal of Medicine
|
November 20, 2014
Long-term safety and efficacy of factor IX gene therapy in hemophilia B
Amit C Nathwani, Ulreke M Reiss, Edward G D Tuddenham, et al.
The New England Journal of Medicine
|
December 14, 2011
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
Amit C Nathwani, Edward G D Tuddenham, Savita Rangarajan, et al.
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of 3