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• ▲ Gerard J. Platenburg

  ProQR Therapeutics (Netherlands) Biochemistry, Genetics and Molecular Biology
  3.47k

  citations

  20

  h-index

  21

  Aachen papers
• ▲ Jim Swildens

  ProQR Therapeutics (Netherlands) Biochemistry, Genetics and Molecular Biology
  565

  citations

  12

  h-index

  13

  Aachen papers
• ▲ Melita Kaltak

  Radboud University Medical Center Biochemistry, Genetics and Molecular Biology
  108

  citations

  5

  h-index

  12

  Aachen papers
• ▲ Willemijn van Leeuwen

  ProQR Therapeutics (Netherlands) Psychology
  27

  citations

  3

  h-index

  1

  Aachen papers

4 results

• Stargardt disease-associated missense and synonymous ABCA4 variants result in aberrant splicing

  2023 Human Molecular Genetics article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access

  10

  citations
• QR-1011 restores defective ABCA4 splicing caused by multiple severe ABCA4 variants underlying Stargardt disease

  2024 Scientific Reports article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access

  8

  citations

2 results