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CELL & GENE THERAPY
INSIGHTS
Rare Diseases – Clinical & Commercial Developments
Guest Editor:
Jaap Boelens, Pediatrician-Oncologist, Immunologist at University Medical Centre Utrecht
Jaap Boelens
Pediatrician-Oncologist, Immunologist at University Medical Centre Utrecht
July 2018
Advances & challenges in developing gene therapies for rare kidney diseases
V Padovano,
M Caplan
4 January 2019
Expert Insight
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Gene Therapy for Childhood Cerebral Adrenoleukodystrophy
C Duncan
17 October 2018
Expert Insight
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SR-TIGET: Continuing to Drive Enabling Technology Development & Clinical Innovation in Rare Diseases
A Aiuti
17 October 2018
Interview
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The Critical Role of Patient Advocacy Groups in the Development of Rare Disease Gene Therapies
S Wilson,
E Linton
16 October 2018
Interview
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Unique Role of Patient Advocacy Groups in Clinical Development of CGTx for Rare Diseases
M Berg
16 October 2018
Editorial
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The Future of Gene Therapy for Rare Eye Diseases
A Girach
16 October 2018
Interview
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Leukodystrophies: a Patient Advocacy Perspective
M Kefalas
16 October 2018
Interview
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Gene replacement therapy for hemoglobinopathies: clinical benefit & challenges for widespread utilization
M Cavazzana,
A Miccio,
I Andre-Schmutz
et al.
16 October 2018
Expert Insight
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Advancing the Development of AAV-Based Gene Therapy for Rare Diseases
S Prasad
14 October 2018
Interview
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Developing Gene Therapy for CEP290-Associated Retinal Ciliopathies
H Khanna
2 October 2018
Editorial
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