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Emerging Gene Therapies – Trends within the Technological, Clinical, Regulatory and Competitive Landscape

  • Published: Jan-2019
  • Report Code: GBI095CBR
  • Report Format: pdf

Description

List of Figures

Figure 1: Nucleases Based on Protein-DNA Interactions

Figure 2: Transcription Activator-Like Effector Nucleases (TALENs)

Figure 3: The CRISPR/Cas System

Figure 4: CRISPR/Cas Binding Mechanism

Figure 5: Cas9 Orthologs

Figure 6: Gene Therapy Clinical Trials Worldwide by Vector

Figure 7: Multiple Sangamo Therapeutics Products Use an Albumin Targeting ‘Safe Harbor’ Approach

Figure 8: Gene Editing Landscape, Progression of Gene Therapy Applications

Figure 9: Gene Editing Landscape, Hemophilia A + B

Figure 10: SPK-9001: Factor IX Activity after SPK-9011 in 8 Participants that did not Show AAV Capsid-Directed Immune Response

Figure 11: Cost of Beta-Thalassemia Major Treatment as of NHS Tariffs at 2013/14 Prices

Figure 12: BCL11A is Involved in Fetal Hemoglobin Silencing

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