Sharif-Askari, Narjes Saheb Sharif-Askari, Fatemeh Saheb Mdkhana, Bushra Al Heialy, Saba Alsafar, Habiba S Hamoudi, Rifat Hamid, Qutayba Halwani, Rabih
Published in
Molecular therapy. Methods & clinical development
The immune system is tightly regulated by the activity of stimulatory and inhibitory immune receptors. This immune homeostasis is usually disturbed during chronic viral infection. Using publicly available transcriptomic datasets, we conducted an in-silico analyses to evaluate the expression pattern of 38 selected immunoinhibitory receptors (IRs) as...
Riberdy, Janice M Zhou, Sheng Zheng, Fei Kim, Young-In Moore, Jennifer Vaidya, Abishek Throm, Robert E Sykes, April Sahr, Natasha Bonifant, Challice L
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Published in
Molecular therapy. Methods & clinical development
Chimeric antigen receptor (CAR) T cells targeting CD123, an acute myeloid leukemia (AML) antigen, hold the promise of improving outcomes for patients with refractory/recurrent disease. We generated five lentiviral vectors encoding CD20, which may serve as a target for CAR T cell depletion, and 2nd or 3rd generation CD123-CARs since the benefit of t...
Khoshnejad, Makan Perales-Puchalt, Alfredo Dia, Yaya Xiao, Peng Patel, Ami Xu, Ziyang Zhu, Xizhou Yun, Kun Baboo, Ishana Qureshi, Rehman
...
Published in
Molecular therapy. Methods & clinical development
Arginase is a complex and unique enzyme that plays diverse roles in health and disease. By metabolizing arginine, it can shape the outcome of innate and adaptive immune responses. The immunomodulatory capabilities of arginase could potentially be applied for local immunosuppression or induction of immune tolerance. With the use of an enhanced DNA d...
Wang, Qingnan Zhong, Xiaomei Li, Qian Su, Jing Liu, Yi Mo, Li Deng, Hongxin Yang, Yang
Published in
Molecular therapy. Methods & clinical development
Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 loaded by vectors could induce high rates of specific site genome editing and correct disease-causing mutations. However, most monogenic genetic diseases such as hemophilia are caused by different mutations dispersed in one gene, instead of an accordant mutation. Vectors develo...
Fong, Sylvia Handyside, Britta Sihn, Choong-Ryoul Liu, Su Zhang, Lening Xie, Lin Murphy, Ryan Galicia, Nicole Yates, Bridget Minto, Wesley C
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Published in
Molecular therapy. Methods & clinical development
Adeno-associated virus 5 (AAV5)-human factor VIII-SQ (hFVIII-SQ; valoctocogene roxaparvovec) is an AAV-mediated product under evaluation for treatment of severe hemophilia A, which contains a B-domain-deleted hFVIII (hFVIII-SQ) transgene and a hybrid liver-specific promotor (HLP). To increase FVIII-SQ expression and reduce the vector dose required,...
Ghassemi, Saba Martinez-Becerra, Francisco J Master, Alyssa M Richman, Sarah A Heo, David Leferovich, John Tu, Yitao García-Cañaveras, Juan Carlos Ayari, Asma Lu, Yinan
...
Published in
Molecular therapy. Methods & clinical development
Effective chimeric antigen receptor (CAR)-T cell therapy is dependent on optimal cell culture methods conducive to the activation and expansion of T cells ex vivo, as well as infection with CAR. Media formulations used in CAR-T cell manufacturing have not been optimized for gene delivery, cell expansion, and overall potency. Bioactive components an...
Yang, Chun Tian, Wenhong Ma, Sisi Guo, Mengmeng Lin, Xiao Gao, Fengying Dong, Xiaoyan Gao, Mingming Wang, Yuhui Liu, George
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Published in
Molecular therapy. Methods & clinical development
Apolipoprotein C2 (ApoC2) is a key activator of lipoprotein lipase for plasma triglyceride metabolism. ApoC2-deficient patients present with severe hypertriglyceridemia and recurrent acute pancreatitis, for whom the only effective treatment is the infusion of normal plasma containing ApoC2. However, since ApoC2 has a fast catabolic rate, a repeated...
Ernst, Martijn P T Broeders, Mike Herrero-Hernandez, Pablo Oussoren, Esmee van der Ploeg, Ans T Pijnappel, W W M Pim
Published in
Molecular therapy. Methods & clinical development
We present an overview of clinical trials involving gene editing using clustered interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription activator-like effector nucleases (TALENs), or zinc finger nucleases (ZFNs) and discuss the underlying mechanisms. In cancer immunotherapy, gene editing is applied ex vivo ...
Radtke, Stefan Pande, Dnyanada Cui, Margaret Perez, Anai M Chan, Yan-Yi Enstrom, Mark Schmuck, Stefanie Berger, Andrew Eunson, Tom Adair, Jennifer E
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Published in
Molecular therapy. Methods & clinical development
Hematopoietic stem cell (HSC) gene therapy has the potential to cure many genetic, malignant, and infectious diseases. We have shown in a nonhuman primate gene therapy and transplantation model that the CD34+CD90+ cell fraction was exclusively responsible for multilineage engraftment and hematopoietic reconstitution. In this study, we show the tran...
Xu, Yinxia Guo, Ping Zhang, Junping Chrzanowski, Matthew Chew, Helen Firrman, Jenni A Sang, Nianli Diao, Yong Xiao, Weidong
Published in
Molecular therapy. Methods & clinical development
Physical titers for recombinant adeno-associated viral (rAAV) vectors are measured by quantifying viral genomes. It is generally perceived that AAV virions disassemble and release DNA upon thermal treatment. Here, we present data on enzymatic accessibility of rAAV genomes when AAV virions were subjected to thermal treatment. For rAAV vectors with a...
