Our investigation into FAM161A gene replacement for RP28 emphasizes the importance of precise therapeutic gene regulation, appropriate vector dosing, and delivery of both isoforms. This precision is pivotal for secure gene therapy involving structural proteins like FAM161A....
CDS-R03066-1 pDonR223-FAM161A GateWay 2ug/0.5ml ￥0 现货 CDS-R03066-2 pMD18-T-FAM161A TA克隆 2ug/0.5ml ￥0 1-2周FAM161A 重组腺病毒 货号产品名称Method规格价格货期 CDS-R03066-11 rAd-FAM161A 重组过表达腺病毒 10^10 vg/支 ￥2500 3-5周 CDS-R03066-12 rAd-sh-FAM161A 重组干扰...
Purpose : Gene replacement is the most intuitive approach to recessive diseases, and has spearheaded the development of gene therapy. Defects in the FAM161A gene encoding for two isoforms of a ciliary protein have been proven to cause autosomal recessive retinitis pigmentosa (RP28). FAM161A is ...
Mutations in the FAM161A gene have been reported in association with autosomal recessive retinitis pigmentosa (arRP) in several ethnic populations. This study aimed to assess the prevalence of FAM161A-related retinopathy in a British cohort and to characterise the phenotype associated with mutations ...
货号产品名称Method规格价格货期 CDS-M03079-11rAd-FAM161A重组腺病毒10^10cfu/支￥30003-5周 CDS-M03079-12rAAV-FAM161A重组腺相关病毒10^11cfu/支￥30003-5周 CDS-M03079-13rLV-FAM161A重组慢病毒10^8TU/支￥30003-5周 FAM161A ORF克隆 ...
We have previously demonstrated that RP can be caused by recessive mutations in the human FAM161A gene, encoding a protein with unknown function that contains a conserved region shared only with a distant paralog, FAM161B. In this study, we show that FAM161A localizes at the base of the ...
Purpose: We have previously demonstrated that mutations in the FAM161A gene, encoding a protein with unknown function and no similarities with other characterized sequences, cause autosomal recessive retinitis pigmentosa (RP). The purpose of this work i...
The median age of disease onset for PRA in the six ESD cases is 4.99 years, which is comparable to Tibetan Terrier and Tibetan Spaniels affected by PRA3, breeds in which the causal PRA variant is also located in the FAM161A gene [3]. There was one outlier in the ESD-affected cohort...
Mutations in the FAM161A gene have been reported in association with autosomal recessive retinitis pigmentosa (arRP) in several ethnic populations. This study aimed to assess the prevalence of FAM161A-related retinopathy in a British cohort and to characterise the phenotype associated with mutations ...
Therefore, the KI mouse model can serve as a suitable model to test this therapeutic modality in vivo as well as other modalities such as gene augmentation therapy and RNA editing.Prakadeeswari GopalakrishnanChen MatsevichEyal BaninDror Sharon...