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molecular therapy methods & clinical development

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Indexings
ncbi
Short Name mol ther methods clin dev
Abbreviated Name mol ther methods clin dev
ISSN 2329-0501
Aim and Scope
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Publications Per Year

Recent Articles (5 total)

2019
AAV-Mediated Expression of Broadly Neutralizing and Vaccine-like Antibodies Targeting the HIV-1 Envelope V2 Region.
van den Berg, Fiona T;Makoah, Nigel A;Ali, Stuart A;Scott, Tristan A;Mapengo, Rutendo E;Mutsvunguma, Lorraine Z;Mkhize, Nonhlanhla N;Lambson, Bronwen E;Kgagudi, Prudence D;Crowther, Carol;Abdool Karim, Salim S;Balazs, Alejandro B;Weinberg, Marc S;Ely, Abdullah;Arbuthnot, Patrick B;Morris, Lynn;
Vol. 14 , pp. 100-112
285 views DOI Abstract
2020
Novel Variants and Mosaicism in Pompe Disease Identified by Extended Analyses of Patients with an Incomplete DNA Diagnosis.
In 't Groen, Stijn L M;de Faria, Douglas O S;Iuliano, Alessandro;van den Hout, Johanna M P;Douben, Hannie;Dijkhuizen, Trijnie;Cassiman, David;Witters, Peter;Barba Romero, Miguel-Ángel;de Klein, Annelies;Somers-Bolman, Galhana M;Saris, Jasper J;Hoefsloot, Lies H;van der Ploeg, Ans T;Bergsma, Atze J;Pijnappel, W W M Pim;
Vol. 17 , pp. 337-348
252 views DOI Abstract
2019
RNA Virus-Based Episomal Vector with a Fail-Safe Switch Facilitating Efficient Genetic Modification and Differentiation of iPSCs.
Komatsu, Yumiko;Takeuchi, Dan;Tokunaga, Tomoya;Sakurai, Hidetoshi;Makino, Akiko;Honda, Tomoyuki;Ikeda, Yasuhiro;Tomonaga, Keizo;
Vol. 14 , pp. 47-55
247 views DOI Abstract
2020
Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease.
Han, Sang-Oh;Li, Songtao;McCall, Angela;Arnson, Benjamin;Everitt, Jeffrey I;Zhang, Haoyue;Young, Sarah P;ElMallah, Mai K;Koeberl, Dwight D;
Vol. 17 , pp. 133-142
216 views DOI Abstract
2019
Induction of Expandable Tissue-Specific Progenitor Cells from Human Pancreatic Tissue through Transient Expression of Defined Factors.
Noguchi, Hirofumi;Miyagi-Shiohira, Chika;Nakashima, Yoshiki;Kinjo, Takao;Kobayashi, Naoya;Saitoh, Issei;Watanabe, Masami;Shapiro, A M James;Kin, Tatsuya;
Vol. 13 , pp. 243-252
213 views DOI Abstract
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