Journal: International Journal of Molecular Sciences

Article Title: Gene Therapy for Achromatopsia

doi: 10.3390/ijms25179739

Figure Lengend Snippet: Summary of clinical trials for gene therapy in Achromatopsia.

Article Snippet: 03758404 03278873 , I/II , CNGA3 , AAV5 , AAV2/8-hG1.7p.co CNGA3 , MeiraGTx/ Janssen , Completed; active , Subretinal.

Techniques: Plasmid Preparation

Journal: International Journal of Molecular Sciences

Article Title: Ocular Therapeutics and Molecular Delivery Strategies for Neovascular Age-Related Macular Degeneration (nAMD)

doi: 10.3390/ijms221910594

Figure Lengend Snippet: Completed and ongoing gene therapy clinical trials for nAMD. Adapted from [ 182 ].

Article Snippet: PEDF , AAV5 , I , Intravitreal , Completed , GenVec , NCT00109499.

Techniques: Plasmid Preparation

Journal: Analytical biochemistry

Article Title: Separation of full and empty adeno-associated virus capsids by anion-exchange chromatography using choline-type salts.

doi: 10.1016/j.ab.2023.115421

Figure Lengend Snippet: Fig. 2. Separation of 50:50 mixtures of AAV5 full and empty capsids using different types of quaternary ammonium salts and NaCl control in a Q-STAT AEX column. Salt gradients used are listed above each elution chromatogram. Betaine without NaCl did not elute AAV5 from the column.

Article Snippet: AAV2, AAV5, AAV6, and AAV8 samples, with or without ssDNA payload of 4.7 kb (here viral capsids denoted as “full” or “empty”, respectively), were obtained from Virovek (Hayward, CA, USA) at a concentration of 2.0 × 1013 vc/mL.

Techniques: Control

Journal: Analytical biochemistry

Article Title: Separation of full and empty adeno-associated virus capsids by anion-exchange chromatography using choline-type salts.

doi: 10.1016/j.ab.2023.115421

Figure Lengend Snippet: Fig. 3. Empty and full AAV5 capsid ratios determined using a choline-Cl gradient in a Q-STAT AEX column. Percent mixtures of AAV5 empty and full capsids were prepared as listed in each elution chromatogram according to supplier’s direction. A choline-Cl gradient (100–350 mM) was applied for elution of empty and full capsids and a fluorescence signal was used for detection. Short red arrows point to minor impurities present in 100 % empty and 100 % full viral capsid stocks.

Article Snippet: AAV2, AAV5, AAV6, and AAV8 samples, with or without ssDNA payload of 4.7 kb (here viral capsids denoted as “full” or “empty”, respectively), were obtained from Virovek (Hayward, CA, USA) at a concentration of 2.0 × 1013 vc/mL.

Techniques: Fluorescence

Journal: Analytical biochemistry

Article Title: Separation of full and empty adeno-associated virus capsids by anion-exchange chromatography using choline-type salts.

doi: 10.1016/j.ab.2023.115421

Figure Lengend Snippet: Fig. 4. Linearity and limit of detection determination. (A) A mixture of 50 % full and 50 % empty capsids of AAV5 was prepared, and serial one to four dilutions from this concentration were made. Each sample (loads listed above each elution chromatogram) was assayed on a Q-STAT AEX column. A choline-Cl gradient (100–350 mM) was applied for elution of capsids, and a fluorescence signal was used for detection. (B) The peak areas of the empty and full samples in relative fluorescence units (RFU counts) were plotted against the number of capsids loaded. (C) Enlarged elution chromatogram for AAV5 mix at the lowest injection amount (3.9 × 108

Article Snippet: AAV2, AAV5, AAV6, and AAV8 samples, with or without ssDNA payload of 4.7 kb (here viral capsids denoted as “full” or “empty”, respectively), were obtained from Virovek (Hayward, CA, USA) at a concentration of 2.0 × 1013 vc/mL.

Techniques: Concentration Assay, Fluorescence, Injection