BACKGROUND: Attempts at gene therapy for the pulmonary manifestations of Cystic Fibrosis have relied mainly on airway delivery. However the efficiency of gene transfer and expression in the airway epi

PubMed Central2002-04-03 更新30

WGS of CCR5 targeting by CRISPR-Cas9 in mauritian cynomolgus macaque blastomeres

基因编辑评估

非人灵长类胚胎模型

The objective was to evaluate on- and off-target editing outcomes in CCR5 CRISPR-Cas9-targeted Mauritian cynomolgus macaque embryos. DNA isolated from individual blastomeres of two embryos, along with

NIAID Data Ecosystem10

Reduction of Natural Adenovirus Tropism to the Liver by both Ablation of Fiber-Coxsackievirus and Adenovirus Receptor Interaction and Use of Replaceable Short Fiber

腺病毒载体

基因治疗递送

The initial recognition and binding of adenovirus vector to the host cell surface is mediated by interaction between the adenovirus fiber knob protein and its receptor, the coxsackievirus and adenovir

PubMed Central40

Padlock oligonucleotides for duplex DNA based on sequence-specific triple helix formation

双链DNA检测

基因治疗递送

An oligonucleotide was circularized around double-stranded DNA thanks to triple helix formation. Short oligonucleotides are known to be able to form DNA triple helices by binding into the DNA major gr

PubMed Central1999-09-14 更新30

All-in-One Adeno-associated Virus Delivery of NmeCas9

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