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Volume 18
Main
Molecular Therapy — Methods & Clinical Development
Volume 18
Molecular Therapy — Methods & Clinical Development
Volume 18
1
Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors
Rumachik, Neil G.
,
Malaker, Stacy A.
,
Poweleit, Nicole
,
Maynard, Lucy H.
,
Adams, Christopher M.
,
Leib, Ryan D.
,
Cirolia, Giana
,
Thomas, Dennis
,
Stamnes, Susan
,
Holt, Kathleen
,
Sinn, Patrick
,
May, Andr
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 5.13 MB
Vos balises:
2020
2
Circulating Biomarkers in Muscular Dystrophies: Disease and Therapy Monitoring
Koutsoulidou, Andrie
,
Phylactou, Leonidas A.
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 724 KB
Vos balises:
2020
3
Role of Essential Metal Ions in AAV Vector-Mediated Transduction
Rambhai, Himanshu K.
,
Ashby, Frederick J.
,
Qing, Keyun
,
Srivastava, Arun
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.34 MB
Vos balises:
2020
4
The Use of Values WNR and GNR to Distinguish between and Diagnose Different Types of Pancreatitis
Luo, Liwen
,
Zhang, Junfeng
,
Yang, Jiali
,
Zhang, Hongyu
,
Tang, Yichen
,
Yang, Di
,
Dong, Hui
,
Wu, Yuzhang
,
Wang, Huaizhi
,
Ni, Bing
,
Tian, Zhiqiang
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 893 KB
Vos balises:
2020
5
Administration of Slow-Release Synthetic Prostacyclin Agonist Promoted Angiogenesis and Skeletal Muscle Regeneration for Limb Ischemia
Nakagawa, Takaya
,
Miyagawa, Shigeru
,
Shibuya, Takashi
,
Sakai, Yoshiki
,
Harada, Akima
,
Watanabe, Kenichi
,
Sawa, Yoshiki
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.65 MB
Vos balises:
2020
6
rAAV8 and rAAV9-Mediated Long-Term Muscle Transduction with Tacrolimus (FK506) in Non-Human Primates
Ishii, Akiko
,
Okada, Hironori
,
Hayashita-Kinoh, Hiromi
,
Shin, Jin-Hong
,
Tamaoka, Akira
,
Okada, Takashi
,
Takeda, Shinâichi
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 999 KB
Vos balises:
2020
7
Systemic Delivery of AAV-Fdxr Mitigates the Phenotypes of Mitochondrial Disorders in Fdxr Mutant Mice
Yang, Li
,
Slone, Jesse
,
Zou, Weiwei
,
Queme, Luis F.
,
Jankowski, Michael P.
,
Yin, Fei
,
Huang, Taosheng
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 4.78 MB
Vos balises:
2020
8
Comparison of AAV-Mediated Optogenetic Vision Restoration between Retinal Ganglion Cell Expression and ON Bipolar Cell Targeting
Lu, Qi
,
Ganjawala, Tushar H.
,
Krstevski, Andrea
,
Abrams, Gary W.
,
Pan, Zhuo-Hua
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.01 MB
Vos balises:
2020
9
Ocular Tolerability and Immune Response to Corneal Intrastromal AAV-IDUA Gene Therapy in New Zealand White Rabbits
Song, Liujiang
,
Bower, Jacquelyn J.
,
Llanga, Telmo
,
Salmon, Jacklyn H.
,
Hirsch, Matthew L.
,
Gilger, Brian C.
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.65 MB
Vos balises:
2020
10
Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine Model
Sawamoto, Kazuki
,
Karumuthil-Melethil, Subha
,
Khan, Shaukat
,
Stapleton, Molly
,
Bruder, Joseph T.
,
Danos, Olivier
,
Tomatsu, Shunji
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.81 MB
Vos balises:
2020
11
Evaluating the Potential of T Cell Receptor Repertoires in Predicting the Prognosis of Resectable Non-Small Cell Lung Cancers
Song, Zhengbo
,
Chen, Xiangbin
,
Shi, Yi
,
Huang, Rongfang
,
Wang, Wenxian
,
Zhu, Kunshou
,
Lin, Shaofeng
,
Wang, Minxian
,
Tian, Geng
,
Yang, Jialiang
,
Chen, Gang
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.29 MB
Vos balises:
2020
12
Unexpected Mutations by CRISPR-Cas9 CTG Repeat Excision in Myotonic Dystrophy and Use of CRISPR Interference as an Alternative Approach
Ikeda, Miki
,
Taniguchi-Ikeda, Mariko
,
Kato, Takema
,
Shinkai, Yasuko
,
Tanaka, Sonoko
,
Hagiwara, Hiroki
,
Sasaki, Naomichi
,
Masaki, Toshihiro
,
Matsumura, Kiichiro
,
Sonoo, Masahiro
,
Kurahashi, Hiroki
,
Sai
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.50 MB
Vos balises:
2020
13
Circular RNAs: Promising Molecular Biomarkers of Human Aging-Related Diseases via Functioning as an miRNA Sponge
Ren, Shuyue
,
Lin, Peirong
,
Wang, Jingrong
,
Yu, Haoying
,
Lv, Tingting
,
Sun, Lan
,
Du, Guanhua
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.10 MB
Vos balises:
2020
14
Characteristics of the Coronavirus Disease 2019 and related Therapeutic Options
Huang, Boxuan
,
Ling, Rongsong
,
Cheng, Yifan
,
Wen, Jieqi
,
Dai, Yarong
,
Huang, Wenjie
,
Zhang, Siyan
,
Lu, Xifeng
,
Luo, Yifeng
,
Jiang, Yi-Zhou
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.16 MB
Vos balises:
2020
15
The Two Faces of ACE2: The Role of ACE2 Receptor and Its Polymorphisms in Hypertension and COVID-19
Bosso, Mira
,
Thanaraj, Thangavel Alphonse
,
Abu-Farha, Mohamed
,
Alanbaei, Muath
,
Abubaker, Jehad
,
Al-Mulla, Fahd
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 549 KB
Vos balises:
2020
16
Protein Delivery of Cell-Penetrating Zinc-Finger Activators Stimulates Latent HIV-1-Infected Cells
Perdigão, Pedro R.L.
,
Cunha-Santos, Catarina
,
Barbas, Carlos F.
,
Santa-Marta, Mariana
,
Goncalves, Joao
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.07 MB
Vos balises:
2020
17
Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease
Meena, Naresh Kumar
,
Ralston, Evelyn
,
Raben, Nina
,
Puertollano, Rosa
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 3.20 MB
Vos balises:
2020
18
Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice
Zou, Chenhui
,
Vercauteren, Koen O.A.
,
Michailidis, Eleftherios
,
Kabbani, Mohammad
,
Zoluthkin, Irene
,
Quirk, Corrine
,
Chiriboga, Luis
,
Yazicioglu, Mustafa
,
Anguela, Xavier M.
,
Meuleman, Philip
,
High, K
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.26 MB
Vos balises:
2020
19
Overcoming Compensatory Mechanisms toward Chronic Drug Administration to Ensure Long-Term, Sustainable Beneficial Effects
Ilan, Yaron
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 350 KB
Vos balises:
2020
20
Analysis of Aflibercept Expression in NHPs following Intravitreal Administration of ADVM-022, a Potential Gene Therapy for nAMD
Kiss, Szilárd
,
Grishanin, Ruslan
,
Nguyen, Aivan
,
Rosario, Romeo
,
Greengard, Judith S.
,
Nieves, Julio
,
Gelfman, Claire M.
,
Gasmi, Mehdi
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 901 KB
Vos balises:
2020
21
Transient Chimeric Ad5/37 Fiber Enhances NK-92 Carrier Cell-Mediated Delivery of Oncolytic Adenovirus Type 5 to Tumor Cells
Gao, Jian
,
Zhang, Wenli
,
Mese, Kemal
,
Bunz, Oskar
,
Lu, Fengmin
,
Ehrhardt, Anja
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.52 MB
Vos balises:
2020
22
Efficient CRISPR-Cas9-Mediated Gene Ablation in Human Keratinocytes to Recapitulate Genodermatoses: Modeling of Netherton Syndrome
Gálvez, Victoria
,
Chacón-Solano, Esteban
,
Bonafont, Jose
,
MencÃa, Ãngeles
,
Di, Wei-Li
,
Murillas, Rodolfo
,
Llames, Sara
,
Vicente, Asunción
,
Del Rio, Marcela
,
Carretero, Marta
,
Larcher, Fernando
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 4.38 MB
Vos balises:
2020
23
Development of AAV Variants with Human Hepatocyte Tropism and Neutralizing Antibody Escape Capacity
Pei, Xiaolei
,
Shao, Wenwei
,
Xing, Allene
,
Askew, Charles
,
Chen, Xiaojing
,
Cui, Caibin
,
Abajas, Yasmina L.
,
Gerber, David A.
,
Merricks, Elizabeth P.
,
Nichols, Timothy C.
,
Li, Wuping
,
Samulski, R. Jude
,
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.12 MB
Vos balises:
2020
24
Effects of Thermally Induced Configuration Changes on rAAV Genomeâs Enzymatic Accessibility
Xu, Yinxia
,
Guo, Ping
,
Zhang, Junping
,
Chrzanowski, Matthew
,
Chew, Helen
,
Firrman, Jenni A.
,
Sang, Nianli
,
Diao, Yong
,
Xiao, Weidong
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.14 MB
Vos balises:
2020
25
Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy
Mariot, Virginie
,
Le Guiner, Caroline
,
Barthélémy, Inès
,
Montus, Marie
,
Blot, Stéphane
,
Torelli, Silvia
,
Morgan, Jennifer
,
Muntoni, Francesco
,
Voit, Thomas
,
Dumonceaux, Julie
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 717 KB
Vos balises:
2020
26
AAV-Mediated Combination Gene Therapy for Neuropathic Pain: GAD65, GDNF, and IL-10
Kim, Daewook
,
Kim, Kyung-Ran
,
Kwon, Yejin
,
Kim, Minjung
,
Kim, Min-Ju
,
Sim, Yeomoon
,
Ji, Hyelin
,
Park, Jang-Joon
,
Cho, Jong-Ho
,
Choi, Heonsik
,
Kim, Sujeong
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.00 MB
Vos balises:
2020
27
Liver-Specific siRNA-Mediated Stat3 or C3 Knockdown Improves the Outcome of Experimental Autoimmune Myocarditis
Avalle, Lidia
,
Marino, Francesca
,
Camporeale, Annalisa
,
Guglielmi, Chiara
,
Viavattene, Daniele
,
Bandini, Silvio
,
Conti, Laura
,
Cimino, James
,
Forni, Marco
,
Zanini, Cristina
,
Ghigo, Alessandra
,
Bogorad
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 3.32 MB
Vos balises:
2020
28
A Novel Gene Therapy Approach for GSD III Using an AAV Vector Encoding a Bacterial Glycogen Debranching Enzyme
Lim, Jeong-A
,
Choi, Su Jin
,
Gao, Fengqin
,
Kishnani, Priya S.
,
Sun, Baodong
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.49 MB
Vos balises:
2020
29
Adenovirus-Mediated LAMA3 Transduction Enhances Hemidesmosome Formation and Periodontal Reattachment during Wound Healing
Li, Yongzheng
,
Zhang, Jing
,
Cheng, Zhenxuan
,
Wang, Ying
,
Huang, Tingben
,
Lai, Kaichen
,
Du, Xue
,
Jiang, Zhiwei
,
Yang, Guoli
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 6.17 MB
Vos balises:
2020
30
A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene
Aronson, Sem J.
,
Bakker, Robert S.
,
Moenis, Sascha
,
van Dijk, Remco
,
Bortolussi, Giulia
,
Collaud, Fanny
,
Shi, Xiaoxia
,
Duijst, Suzanne
,
ten Bloemendaal, Lysbeth
,
Ronzitti, Giuseppe
,
Muro, Andrés F.
,
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 896 KB
Vos balises:
2020
31
Intramuscular Delivery of Replicon RNA Encoding ZIKV-117 Human Monoclonal Antibody Protects against Zika Virus Infection
Erasmus, Jesse H.
,
Archer, Jacob
,
Fuerte-Stone, Jasmine
,
Khandhar, Amit P.
,
Voigt, Emily
,
Granger, Brian
,
Bombardi, Robin G.
,
Govero, Jennifer
,
Tan, Qing
,
Durnell, Lorellin A.
,
Coler, Rhea N.
,
Diamond
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.39 MB
Vos balises:
2020
32
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction
Yasuda, Makiko
,
Huston, Marshall W.
,
Pagant, Silvere
,
Gan, Lin
,
St. Martin, Susan
,
Sproul, Scott
,
Richards, Daniel
,
Ballaron, Stephen
,
Hettini, Khaled
,
Ledeboer, Annemarie
,
Falese, Lillian
,
Cao, Lichi
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.03 MB
Vos balises:
2020
33
Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter
Fong, Sylvia
,
Handyside, Britta
,
Sihn, Choong-Ryoul
,
Liu, Su
,
Zhang, Lening
,
Xie, Lin
,
Murphy, Ryan
,
Galicia, Nicole
,
Yates, Bridget
,
Minto, Wesley C.
,
Vitelli, Catherine
,
Harmon, Danielle
,
Ru, Yuanbi
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.36 MB
Vos balises:
2020
34
The Art and Science of Selecting a CD123-Specific Chimeric Antigen Receptor for Clinical Testing
Riberdy, Janice M.
,
Zhou, Sheng
,
Zheng, Fei
,
Kim, Young-In
,
Moore, Jennifer
,
Vaidya, Abishek
,
Throm, Robert E.
,
Sykes, April
,
Sahr, Natasha
,
Bonifant, Challice L.
,
Ryu, Byoung
,
Gottschalk, Stephen
,
Ve
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.52 MB
Vos balises:
2020
35
CRISPR-Cas9-Mediated In Vivo Gene Integration at the Albumin Locus Recovers Hemostasis in Neonatal and Adult Hemophilia B Mice
Wang, Qingnan
,
Zhong, Xiaomei
,
Li, Qian
,
Su, Jing
,
Liu, Yi
,
Mo, Li
,
Deng, Hongxin
,
Yang, Yang
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.14 MB
Vos balises:
2020
36
Enhancing Chimeric Antigen Receptor T Cell Anti-tumor Function through Advanced Media Design
Ghassemi, Saba
,
Martinez-Becerra, Francisco J.
,
Master, Alyssa M.
,
Richman, Sarah A.
,
Heo, David
,
Leferovich, John
,
Tu, Yitao
,
GarcÃa-Cañaveras, Juan Carlos
,
Ayari, Asma
,
Lu, Yinan
,
Wang, Ai
,
Rabino
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.10 MB
Vos balises:
2020
37
Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65
Hu, Chin-Ju
,
Lu, Ying-Chang
,
Tsai, Yi-Hsiu
,
Cheng, Haw-Yuan
,
Takeda, Hiroki
,
Huang, Chun-Ying
,
Xiao, Ru
,
Hsu, Chuan-Jen
,
Tsai, Jin-Wu
,
Vandenberghe, Luk H.
,
Wu, Chen-Chi
,
Cheng, Yen-Fu
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.51 MB
Vos balises:
2020
38
Identification and Characterization of the Copy Number Dosage-Sensitive Genes in Colorectal Cancer
Chang, Zhiqiang
,
Liu, Xinxin
,
Zhao, Wenyuan
,
Xu, Yan
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.78 MB
Vos balises:
2020
39
Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease
Piras, Giuseppa
,
Montiel-Equihua, Claudia
,
Chan, Yee-Ka Agnes
,
Wantuch, Slawomir
,
Stuckey, Daniel
,
Burke, Derek
,
Prunty, Helen
,
Phadke, Rahul
,
Chambers, Darren
,
Partida-Gaytan, Armando
,
Leon-Rico, Die
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.61 MB
Vos balises:
2020
40
Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease
Hakim, Chady H.
,
Clément, Nathalie
,
Wasala, Lakmini P.
,
Yang, Hsiao T.
,
Yue, Yongping
,
Zhang, Keqing
,
Kodippili, Kasun
,
Adamson-Small, Laura
,
Pan, Xiufang
,
Schneider, Joel S.
,
Yang, N. Nora
,
Chamberl
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 3.67 MB
Vos balises:
2020
41
Design and Testing of Vector-Producing HEK293T Cells Bearing a Genomic Deletion of the SV40Â T Antigen Coding Region
Bae, Dahae Hailey
,
Marino, Michael
,
Iaffaldano, Brian
,
Fenstermaker, Sydney
,
Afione, Sandra
,
Argaw, Takele
,
McCright, Jacob
,
Kwilas, Anna
,
Chiorini, John A.
,
Timmons, Andrew E.
,
Reiser, Jakob
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.08 MB
Vos balises:
2020
42
AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity
Tran, Ngoc Tam
,
Heiner, Cheryl
,
Weber, Kristina
,
Weiand, Michael
,
Wilmot, Daniella
,
Xie, Jun
,
Wang, Dan
,
Brown, Alexander
,
Manokaran, Sangeetha
,
Su, Qin
,
Zapp, Maria L.
,
Gao, Guangping
,
Tai, Phillip W
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.94 MB
Vos balises:
2020
43
Barriers and Strategies of Cationic Liposomes for Cancer Gene Therapy
Liu, Chunyan
,
Zhang, Ligang
,
Zhu, Wenhui
,
Guo, Raoqing
,
Sun, Huamin
,
Chen, Xi
,
Deng, Ning
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.58 MB
Vos balises:
2020
44
Novel Potential Therapeutic Target for E2F1 and Prognostic Factors of E2F1/2/3/5/7/8 in Human Gastric Cancer
Liu, Xuhong
,
Hu, Chunhong
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 6.33 MB
Vos balises:
2020
45
Soluble Klotho Improves Hepatic Glucose and Lipid Homeostasis in Type 2 Diabetes
Gu, Huiying
,
Jiang, Wei
,
You, Nan
,
Huang, Xiaobing
,
Li, Yuming
,
Peng, Xuehui
,
Dong, Rui
,
Wang, Zheng
,
Zhu, Yinan
,
Wu, Ke
,
Li, Jing
,
Zheng, Lu
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 4.70 MB
Vos balises:
2020
46
Use of Nanovesicles from Orange Juice to Reverse Diet-Induced Gut Modifications in Diet-Induced Obese Mice
Berger, Emmanuelle
,
Colosetti, Pascal
,
Jalabert, Audrey
,
Meugnier, Emmanuelle
,
Wiklander, Oscar P.B.
,
Jouhet, Juliette
,
Errazurig-Cerda, Elisabeth
,
Chanon, Stéphanie
,
Gupta, Dhanu
,
Rautureau, Gilles
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 3.33 MB
Vos balises:
2020
47
Simple and Fast SEC-Based Protocol to Isolate Human Plasma-Derived Extracellular Vesicles for Transcriptional Research
Gaspar, Laetitia S.
,
Santana, Magda M.
,
Henriques, Carina
,
Pinto, Maria M.
,
Ribeiro-Rodrigues, Teresa M.
,
Girão, Henrique
,
Nobre, Rui Jorge
,
Pereira de Almeida, LuÃs
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.39 MB
Vos balises:
2020
48
AAV-Mediated ApoC2 Gene Therapy: Reversal of Severe Hypertriglyceridemia and Rescue of Neonatal Death in ApoC2-Deficient Hamsters
Yang, Chun
,
Tian, Wenhong
,
Ma, Sisi
,
Guo, Mengmeng
,
Lin, Xiao
,
Gao, Fengying
,
Dong, Xiaoyan
,
Gao, Mingming
,
Wang, Yuhui
,
Liu, George
,
Xian, Xunde
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 3.09 MB
Vos balises:
2020
49
Rapid In-Process Monitoring of Lentiviral Vector Particles by High-Performance Liquid Chromatography
Transfiguracion, Julia
,
Tran, Michelle Yen
,
Lanthier, Stéphane
,
Tremblay, Sonia
,
Coulombe, Nathalie
,
Acchione, Mauro
,
Kamen, Amine A.
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.26 MB
Vos balises:
2020
50
âPara-retinalâ Vector Administration into the Deep Vitreous Enhances Retinal Transgene Expression
Zeng, Yong
,
Boyd, Ryan
,
Bartoe, Joshua
,
Wiley, Henry E.
,
Marangoni, Dario
,
Wei, Lisa L.
,
Sieving, Paul A.
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 1.77 MB
Vos balises:
2020
51
Generation of Nonhuman Primate Model of Cone Dysfunction through In Situ AAV-Mediated CNGB3 Ablation
Lin, Qiang
,
Lv, Ji-Neng
,
Wu, Kun-Chao
,
Zhang, Chang-Jun
,
Liu, Qin
,
Jin, Zi-Bing
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.21 MB
Vos balises:
2020
52
Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells
Bittorf, Patrick
,
Bergmann, Thorsten
,
Merlin, Simone
,
Olgasi, Cristina
,
Pullig, Oliver
,
Sanzenbacher, Ralf
,
Zierau, Martin
,
Walles, Heike
,
Follenzi, Antonia
,
Braspenning, Joris
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 761 KB
Vos balises:
2020
53
Direct Functional Protein Delivery with a Peptide into Neonatal and Adult Mammalian Inner Ear In Vivo
Zhang, Kun
,
Cheng, Xiaoting
,
Zhao, Liping
,
Huang, Mingqian
,
Tao, Yong
,
Zhang, Hongbo
,
Rosenholm, Jessica M.
,
Zhuang, Min
,
Chen, Zheng-Yi
,
Chen, Bing
,
Shu, Yilai
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 2.56 MB
Vos balises:
2020
54
Ex Vivo Cell Therapy by Ectopic Hepatocyte Transplantation Treats the Porcine Tyrosinemia Model of Acute Liver Failure
Nicolas, Clara T.
,
Kaiser, Robert A.
,
Hickey, Raymond D.
,
Allen, Kari L.
,
Du, Zeji
,
VanLith, Caitlin J.
,
Guthman, Rebekah M.
,
Amiot, Bruce
,
Suksanpaisan, Lukkana
,
Han, Bing
,
Francipane, Maria Giovanna
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 3.52 MB
Vos balises:
2020
55
Purification of Human CD34+CD90+ HSCs Reduces Target Cell Population and Improves Lentiviral Transduction for Gene Therapy
Radtke, Stefan
,
Pande, Dnyanada
,
Cui, Margaret
,
Perez, Anai M.
,
Chan, Yan-Yi
,
Enstrom, Mark
,
Schmuck, Stefanie
,
Berger, Andrew
,
Eunson, Tom
,
Adair, Jennifer E.
,
Kiem, Hans-Peter
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 3.11 MB
Vos balises:
2020
56
CERE-120 Prevents Irradiation-Induced Hypofunction and Restores Immune Homeostasis in Porcine Salivary Glands
Lombaert, Isabelle M.A.
,
Patel, Vaishali N.
,
Jones, Christina E.
,
Villier, Derrick C.
,
Canada, Ashley E.
,
Moore, Matthew R.
,
Berenstein, Elsa
,
Zheng, Changyu
,
Goldsmith, Corinne M.
,
Chorini, John A.
,
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 5.61 MB
Vos balises:
2020
57
A Long Intergenic Non-coding RNA, LINC01426, Promotes Cancer Progression via AZGP1 and Predicts Poor Prognosis in Patients with LUAD
Tian, Baorui
,
Han, Xiaoyang
,
Li, Guanzhen
,
Jiang, Hua
,
Qi, Jianni
,
Li, Jiamei
,
Tian, Yingying
,
Wang, Chuanxi
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 5.57 MB
Vos balises:
2020
58
Synthetic mRNA Encoding VEGF-A in Patients Undergoing Coronary Artery Bypass Grafting: Design of a Phase 2a Clinical Trial
Anttila, Vesa
,
Saraste, Antti
,
Knuuti, Juhani
,
Jaakkola, Pekka
,
Hedman, Marja
,
Svedlund, Sara
,
Lagerström-Fermér, Maria
,
Kjaer, Magnus
,
Jeppsson, Anders
,
Gan, Li-Ming
Journal:
Molecular Therapy — Methods & Clinical Development
Année:
2020
Fichier:
PDF, 797 KB
Vos balises:
2020
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