April 12, 2024

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Recent progress made by scientists in the field of gene therapy research!

Recent progress made by scientists in the field of gene therapy research!

Recent progress made by scientists in the field of gene therapy research!  This article summarizes the recent progress made by scientists in the field of gene therapy and shares with you!

Recent progress made by scientists in the field of gene therapy research!



[1] Science Sub-Journal: Shocked! Clinical trials have shown that gene therapy vector injection into one eye can improve binocular vision

doi: 10.1126/scitranslmed.aaz7423

Leber hereditary optic neuropathy (LHON), also known as Leber optic nerve atrophy, is the most common cause of mitochondrial blindness. In a new clinical study, an international team led by Dr. Patrick Yu-Wai-Man from the University of Cambridge in the UK and Dr. José-Alain Sahel from the Paris Institute of Vision in France injected a gene therapy vector into one eye of a patient with LHON , Can significantly improve the vision of both eyes. The relevant research results were published in the journal Science Translational Medicine.

In this landmark Phase 3 clinical trial, these researchers successfully treated 37 LHON patients. After further clinical trials, this treatment may help thousands of people around the world restore and preserve some of their vision.

The results of these studies indicate that 78% of LHON patients undergoing treatment have significantly improved binocular vision. This suggests that the improvement in vision in the untreated eye may be due to the transfer of viral vector DNA in the eye that received the injection. LHON affects a special type of retinal cells, namely retinal ganglion cells, resulting in optic nerve degeneration and rapid deterioration of binocular vision.

Within a few weeks after the onset of illness, the vision of most affected people will deteriorate to the level of legal blindness. In less than 20% of cases, vision can be restored, and only a few people can achieve more than 20/200 (the largest letter on the standard visual acuity chart). The incidence of LHON is about one in 30,000, most of which are men. Symptoms usually appear in the twenties or thirties.

Most patients have m.11778G>A mutations in the MT-ND4 gene. Existing methods for the treatment of blinding optic neuropathy are still limited.

[2] Curr Gene Ther: Artificial RNA editing based on ADAR is expected to be used in gene therapy

doi: 10.2174/1566523220666200516170137

In a paper recently published in the journal Current Gene Therapy, Tsukahara’s team reviewed the latest research results on the application of ADAR to recover the genetic code, as well as the different methods involved in the use of ADAR for manual RNA editing.

They also talked about comparative studies of various isomers of ADAR. Therefore, they will try to give a detailed overview of the role of artificial RNA editing and ADAR, with a focus on enzymatic targeted A→I editing.

Most artificial RNA editing systems use the active site of the catalytic enzyme ADAR and the guide RNA (gRNA) complementary to the target to recruit the active site to the target RNA. One method of artificial RNA editing is to use chemical methods.

Vogel and colleagues used SNAP tags to link ADAR to gRNA and reported that the system is effective both in vitro and in vivo. However, this technique requires a continuous supply of effector molecules to be effective. It is reported that it can also use RNA-binding protein to bind gRNA to enzymes.

Two tethered systems derived from phage are commonly used in eukaryotes: the Lambda N system and the MS2 system. The use of ADAR enzyme and MS2 system can realize the recovery of the genetic code, which is promising in gene therapy.





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