Featured
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Article |
Activation of homology-directed DNA repair plays key role in CRISPR-mediated genome correction
- Gourish Mondal
- , Caitlin J. VanLith
- & Joseph B. Lillegard
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Review Article |
Prospects of viral vector-mediated delivery of sequences encoding anti-HBV designer endonucleases
- Ridhwaanah Jacobs
- , Prashika Singh
- & Mohube Betty Maepa
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Article |
FEN1 inhibitor synergizes with low-dose camptothecin to induce increased cell killing via the mitochondria mediated apoptotic pathway
- Ting Wu
- , Hongqiao Zhu
- & Zhigang Guo
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Brief Communication
| Open Access
Precise and error-prone CRISPR-directed gene editing activity in human CD34+ cells varies widely among patient samples
- Shirin R. Modarai
- , Sambee Kanda
- & Eric B. Kmiec
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Review Article |
Recent development of AAV-based gene therapies for inner ear disorders
- Yiyang Lan
- , Yong Tao
- & Guisheng Zhong
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Review Article |
Advances in genome editing: the technology of choice for precise and efficient β-thalassemia treatment
- Gibran Ali
- , Muhammad Akram Tariq
- & Javed Akram
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Article |
The deletion of mutant SOD1 via CRISPR/Cas9/sgRNA prolongs survival in an amyotrophic lateral sclerosis mouse model
- Weisong Duan
- , Moran Guo
- & Chunyan Li
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Short Communication |
Variable correction of Artemis deficiency by I-Sce1-meganuclease-assisted homologous recombination in murine hematopoietic stem cells
- J Rivière
- , J Hauer
- & M Cavazzana
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Original Article |
Hairpin-end conformation of adeno-associated virus genome determines interactions with DNA-repair pathways
- M P Cataldi
- & D M McCarty
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Original Article |
Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs
- B L Ellis
- , M L Hirsch
- & M H Porteus
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Original Article |
Investigation of the peak action wavelength of light-activated gene transduction
- M Myakishev-Rempel
- , J Kuper
- & E M Schwarz
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Original Article |
Self-complementary AAV mediates gene targeting and enhances endonuclease delivery for double-strand break repair
- M L Hirsch
- , L Green
- & R J Samulski
Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo
Progress and prospects: oligonucleotide-directed gene modification in mouse embryonic stem cells: a route to therapeutic application