Tom3 schreef op 16 november 2019 11:38:
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Ik blijf rustig zitten in ProQR. Ik moet nog zien of Crispr/CAS9 vlotjes de eindstreep haalt. Als er ook maar 1 geval van een onherstelbare fout optreedt, staat de FDA geheid op de rem. Recentelijk zijn door de FDA 4 gen therapieën on hold gezet. Er zullen nog meer volgen getuige het navolgende fragment uit De Boer zijn update over Axiomer dit voorjaar:
"In addition, CRISPR genome editing creates permanent changes to a person’s genetic code. Many viral vectors that are being used for gene therapy integrate their viral genomes into the host’s DNA. This alters the genome in a permanent fashion. Permanent alterations of the genome, when solely therapeutic, can be positive. But recent evidence has surfaced demonstrating off-target CRISPR editing that suggests there are significant inherent risks to using this technique. Off-target genome editing can disrupt other normally functioning genes and can cause health problems or toxicity of their own. EONs used for Axiomer have high-fidelity for their intended target such that there is a low probability of off-target activity. In addition to the nonpermanent nature of Axiomer, any potential damage that could happen to the RNA will not be permanent."