Wang, Xianqiang Guo, Sen Zhao, Rui Liu, Yanfeng Yang, Guangyun
Published in
Human gene therapy
Hepatoblastoma (HB) usually occurs in infants and toddlers. Although long non-coding RNAs (lncRNAs) in various human cancers have been widely studied, the role of lncRNAs in HB remains unclear. This study aimed to investigate the biological role of the lncRNA lung cancer associated transcript 1 (LUCAT1) in HB. Analysis of data from The Cancer Genom...
McCullough, K Tyler Boye, Sanford L Fajardo, Diego Calabro, Kaitlyn Peterson, James J Strang, Christianne E Chakraborty, Dibyendu Gloskowski, Sebastian Haskett, Scott Samuelsson, Steven
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Published in
Human gene therapy
Mutations in GUCY2D, the gene encoding retinal guanylate cyclase-1 (retGC1), are the leading cause of autosomal dominant cone-rod dystrophy (CORD6). Significant progress toward clinical application of gene replacement therapy for Leber congenital amaurosis (LCA) due to recessive mutations in GUCY2D (LCA1) has been made, but a different approach is ...
Gong, Yi Berenson, Anna Laheji, Fiza Gao, Guangping Wang, Dan Ng, Carrie Volak, Adrienn Kok, Rene Kreouzis, Vasileios Dijkstra, Inge M
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Published in
Human gene therapy
Mutations in the gene encoding the peroxisomal ATP-binding cassette transporter (ABCD1) cause elevations in very long-chain fatty acids (VLCFAs) and the neurodegenerative disease adrenoleukodystrophy (ALD). In most adults, this manifests as the spinal cord axonopathy adrenomyeloneuropathy (AMN). A challenge in virus-based gene therapy in AMN is how...
Yan, Ziying Zou, Wei Feng, Zehua Shen, Weiran Park, Soo Yeun Deng, Xuefeng Qiu, Jianming Engelhardt, John F
Published in
Human gene therapy
The genome of recombinant adeno-associated virus 2 (rAAV2) remains a promising candidate for gene therapy for cystic fibrosis (CF) lung disease, but due to limitations in the packaging capacity and the tropism of this virus with respect to the airways, strategies have evolved for packaging an rAAV2 genome (up to 5.8 kb) into the capsid of human boc...
Chekuri, Anil Sahu, Bhubanananda Chavali, Venkata Ramana Murthy Voronchikhina, Marina Soto-Hermida, Angel Suk, John J Alapati, Akhila N Bartsch, Dirk-Uwe Ayala-Ramirez, Raul Zenteno, Juan C
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Published in
Human gene therapy
Patients harboring homozygous c.498_499insC mutations in MFRP demonstrate hyperopia, microphthalmia, retinitis pigmentosa, retinal pigment epithelial atrophy, variable degrees of foveal edema, and optic disc drusen. The disease phenotype is variable, however, with some patients maintaining good central vision and cone function till late in the dise...
Ziegler, Tilman Ishikawa, Kiyotake Hinkel, Rabea Kupatt, Christian
Published in
Human gene therapy
Recombinant adeno-associated virus vectors (rAAVs) represent a reliable tool for basic and translational research, while rAAVs are also making strides in early clinical trials as vehicles for gene transfer. Their low immunogenicity, tissue tropism, and relative safety due to their low rate of genomic integration represent key features, making rAAVs...
Zhen, Shuai Lu, Jiaojiao Chen, Wei Zhao, Le Li, Xu
Published in
Human gene therapy
Targeted therapy produces objective responses in bladder cancer patients, although the responses can be short. Meanwhile, response rates to immune therapy are lower, but the effects are more durable. Based on these findings, it was hypothesized that urothelial carcinoma associated 1 (UCA1)-targeted therapy could synergize with programmed cell death...
Schug, Christina Sievert, Wolfgang Urnauer, Sarah Müller, Andrea M Schmohl, Kathrin A Wechselberger, Alexandra Schwenk, Nathalie Lauber, Kirsten Schwaiger, Markus Multhoff, Gabriele
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Published in
Human gene therapy
The tumor-homing properties of mesenchymal stem cells (MSC) have led to their development as delivery vehicles for the targeted delivery of therapeutic genes such as the sodium-iodide symporter (NIS) to solid tumors. External beam radiation therapy may represent an ideal setting for the application of engineered MSC-based gene therapy, as tumor irr...
Du, Sichen Ou, Huayuan Cui, Renjie Jiang, Nan Zhang, Meiqin Li, Xiaorong Ma, Jing Zhang, Jin Ma, Duan
Published in
Human gene therapy
Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder caused by mutations in the GBA gene. Enzyme replacement treatment is the most effective therapy available for type 1 GD patients, but it is very expensive and does not improve neurologic outcomes in type 2 and 3 GD patients. This study evaluated the effectiveness of an adeno-...
Zaneveld, Smriti Agrawal Eblimit, Aiden Liang, Qingnan Bertrand, Renae Wu, Nathaniel Liu, Hehe Nguyen, Quynh Zaneveld, Jacques Wang, Keqing Li, Yumei
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Published in
Human gene therapy
Hereditary retinal dystrophy is clinically defined as a broad group of chronic and progressive disorders that affect visual function by causing photoreceptor degeneration. Previously, we identified mutations in the gene encoding receptor expression-enhancing protein 6 (REEP6), in individuals with autosomal recessive retinitis pigmentosa (RP), the m...
