Zobrazeno 1 - 10
of 321
pro vyhledávání: '"MT: RNA/DNA"'
Autor:
Peisen Huang, Jun Xu, Benjamin Keepers, Yifang Xie, David Near, Yangxi Xu, James Rock Hua, Brian Spurlock, Shea Ricketts, Jiandong Liu, Li Wang, Li Qian
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102390- (2024)
Direct cardiac reprogramming of fibroblasts into induced cardiomyocytes (iCMs) can be achieved by ectopic expression of cardiac transcription factors (TFs) via viral vectors. However, risks like genomic mutations, viral toxicity, and immune response
Externí odkaz:
https://doaj.org/article/c769ac982e584f6f89172252efc78bd4
Autor:
Matthias Thomas Ochmann, Csaba Miskey, Lacramioara Botezatu, Nicolás Sandoval-Villegas, Tanja Diem, Zoltán Ivics
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102381- (2024)
The Sleeping Beauty (SB) transposon system is a useful tool for genetic applications, including gene therapy. We discovered a hyperactive variant of the SB100X transposase, called SB200X. This mutant, resulting from a specific amino acid replacement
Externí odkaz:
https://doaj.org/article/b005b5bfee1f43a997a1022249745dd8
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102383- (2024)
Gene editing provides a promising alternative approach that may achieve long-term FVIII expression for hemophilia A (HemA) treatment. In this study, we investigated in vivo correction of a mutant factor VIII (FVIII) gene in HemA mice. We first develo
Externí odkaz:
https://doaj.org/article/b211a9b2d09a41298d639412f26942df
Autor:
Congwen Shao, Qing Liu, Jinchao Xu, Jianxiang Zhang, Chengpeng Zhang, Ye Xin, Yuhua Ye, Bin Lin, Xinhua Zhang, Li Cheng, Xiangmin Xu, Peng Xu
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102371- (2024)
Hemoglobin Constant Spring (Hb CS) is the most common non-deletional and clinically significant α-thalassemic mutation, and it is caused by an anti-termination mutation at the α2-globin gene stop codon. We developed a prime editing strategy for the
Externí odkaz:
https://doaj.org/article/1865a64661dc4bcab7577484b5cdddd4
Autor:
Yuxi Chen, Rui Kang, Yuanling Jiang, Qi Zheng, Yue Yang, Jiaqi Liu, Guanglan Wu, Weijun Zhao, Zhan Li, Chengxiang Peng, Pengfei Zhang, Fei Peng, Qianyi Liu, Sihui Hu, Xiao Luo, Guifang Wu, Kaixin Cui, Junjiu Huang, Yongming Wang, Zhou Songyang, Puping Liang
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102366- (2024)
Compact and adaptable CRISPR-Cas systems enable genome engineering applications in various contexts via high-efficiency delivery. The adeno-associated virus (AAV) is a widely used delivery system. One of the most compact type II-C Cas9 orthologs—Cj
Externí odkaz:
https://doaj.org/article/f39d0b57ff104d259ae44eff29eff18f
Autor:
Samar Z. Rizvi, Wing Suen Chan, Eleonora Maino, Sydney Steiman, Georgiana Forguson, Maya Klepfish, Ronald D. Cohn, Evgueni A. Ivakine
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102356- (2024)
Recent progress in genome editing technologies has catalyzed the generation of sophisticated cell models; however, the precise modeling of copy-number variation (CNV) diseases remains a significant challenge despite their substantial prevalence in th
Externí odkaz:
https://doaj.org/article/16631687f3624b21a3da29b4dc879eca
Autor:
Anila George, Poornasree Sadanandan, Nithin Sam Ravi, B. Vaishnavi, Srujan Marepally, Saravanbhavan Thangavel, Shaji R. Velayudhan, Alok Srivastava, Kumarasamypet M Mohankumar
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102347- (2024)
Recent studies have shown that base editing, even with single-strand breaks, could result in large deletions of the interstitial regions while targeting homologous regions. Several therapeutically relevant genes such as HBG, HBB, CCR5, and CD33 have
Externí odkaz:
https://doaj.org/article/16c6ab9d93df4d0791d211dc6272b842
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102344- (2024)
CRISPR-Cas9-mediated homology-directed repair (HDR) is a versatile platform for creating precise site-specific DNA insertions, deletions, and substitutions. These precise edits are made possible through the use of exogenous donor templates that carry
Externí odkaz:
https://doaj.org/article/88ca151ef922460fbfa0caae60dd7381
Autor:
Irene Vázquez-Domínguez, Mert Öktem, Florian A. Winkelaar, Thai Hoang Nguyen, Anita D.M. Hoogendoorn, Eleonora Roschi, Galuh D.N. Astuti, Raoul Timmermans, Nuria Suárez-Herrera, Ilaria Bruno, Albert Ruiz-Llombart, Joseph Brealey, Olivier G. de Jong, Rob W.J. Collin, Enrico Mastrobattista, Alejandro Garanto
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102345- (2024)
Deep-intronic (DI) variants represent approximately 10%–12% of disease-causing genetic defects in ABCA4-associated Stargardt disease (STGD1). Although many of these DI variants are amenable to antisense oligonucleotide-based splicing-modulation the
Externí odkaz:
https://doaj.org/article/9cd8756ec318431f8076236a1e749258
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102317- (2024)
Spinocerebellar ataxia type 1 (SCA1) is an adult-onset neurodegenerative disease caused by an expansion of the CAG repeat region of the ATXN1 gene. Currently there are no disease-modifying treatments; however, previous work has shown the potential of
Externí odkaz:
https://doaj.org/article/29e2bfbcf05e4ad1ba3e0ed38d7d2a4d