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Vectorology - GEG Tech top picks
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BigField GEG Tech's insight:
Employing a murine model of retinal disease, the scientists demonstrate that ciliary neurotrophic factor (CNTF) confers life-long protection against photoreceptor degeneration. The mechanisms underlying CNTF-mediated neuroprotection were explored through transcriptome analysis, revealing widespread up-regulation of proteolysis inhibitors, which may prevent cellular/extracellular matrix degradation and complement activation in neurodegenerative diseases. 
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BigField GEG Tech's insight:
Horama has raised a second fundraising round that will support its lead candidate, a gene therapy for retinitis pigmentosa, through clinical trials.
HORA-PDE6B is intended to cure retinitis pigmentosa in patients that develop the disease because of a mutation in the PDE6β gene, which amounts to around 5,000 people in the US and Europe. The gene therapy consists of a recombinant adeno-associated virus (AAV) in which the viral DNA is replaced by a functional copy of the PDE6β gene.
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BigField GEG Tech's insight:
The authors found that the targeting of some parts of the intron promoted trans-splicing more efficiently than the targeting of other areas, and that trans-splicing rate could be increased by modifying the replacement sequence. They tested AAV gene transfer in a mouse model of retinitis pigmentosa that expresses a mutant allele of the human rhodopsin gene, and demonstrated the feasability of trans-splicing in vivo.
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Optogenetics may enable mutation-independent, circuit-specific restoration of neuronal function in neurological diseases. Retinitis pigmentosa is a neurodegenerative eye disease where loss of photoreceptors can lead to complete blindness. In a blind patient, the researchers combined intraocular injection of an adeno-associated viral vector encoding ChrimsonR with light stimulation via engineered goggles.