Research Papers

25 of 702 papers

CSV Excel
Quick filters: Open Data Open Code Pre-registered Funding Declared COI Declared Open Access Clear Filters
Necessity of strengthening the current clinical regulatory for companion diagnostics: An institutional comparison of th…
Kang SL; Woo JH; Kim NH; Kwon JY; Kim SM.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
4/6 Transparent
Data Funding Declared
Establishment of UGT1A1-knockout human iPS-derived hepatic organoids for UGT1A1-specific kinetics and toxicity evaluati…
Shintani T; Imamura C; Ueyama-Toba Y; Inui J; Watanabe A; Mizuguchi H.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Selection of viral capsids and promoters affects the efficacy of rescue of <i>Tmprss3</i>-deficient cochlea.
Aaron KA; Pekrun K; Atkinson PJ; Billings SE; Abitbol JM; Lee IA; Eltawil Y; Chen YS; Dong W; Nelson RF; Kay MA; Cheng AG.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
iPSC-derived mesenchymal stem cells attenuate cerebral ischemia-reperfusion injury by inhibiting inflammatory signaling…
Arakawa M; Sakamoto Y; Miyagawa Y; Nito C; Takahashi S; Nitahara-Kasahara Y; Suda S; Yamazaki Y; Sakai M; Kimura K; Okada T.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease.
Xu L; Lahiri P; Skowronski J; Bhatia N; Lattanzi A; Porteus MH.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Lentiviral delivered aflibercept OXB-203 for treatment of neovascular AMD.
Iqball S; Beck DK; Devarajan G; Khoo CP; O'Connor DM; Ellis S; Guzman E; Mitrophanous KA; Lad Y.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
2/6 Transparent
Inducible HEK293 AAV packaging cell lines expressing Rep proteins.
Jalšić L; Lytvyn V; Elahi SM; Hrapovic S; Nassoury N; Chahal PS; Gaillet B; Gilbert R.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Prevention of early-onset cardiomyopathy in <i>Dmd</i> exon 52-54 deletion mice by CRISPR-Cas9-mediated exon skipping.
Rok M; Wong TWY; Maino E; Ahmed A; Yang G; Hyatt E; Lindsay K; Fatehi S; Marks R; Delgado-Olguín P; Ivakine EA; Cohn RD.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
A method for polyclonal antigen-specific T cell-targeted genome editing (TarGET) for adoptive cell transfer application…
Palianina D; Di Roberto RB; Castellanos-Rueda R; Schlatter F; Reddy ST; Khanna N.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
4/6 Transparent
Data Funding Declared
The p53 challenge of hematopoietic stem cell gene editing.
Dorset SR; Bak RO.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Starburst amacrine cells amplify optogenetic visual restoration through gap junctions.
Katada Y; Kunimi H; Serizawa N; Lee D; Kobayashi K; Negishi K; Okano H; Tanaka KF; Tsubota K; Kurihara T.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
CRISPR-Cas9 correction of a nonsense mutation in <i>LCA5</i> rescues lebercilin expression and localization in human re…
Afanasyeva TAV; Athanasiou D; Perdigao PRL; Whiting KR; Duijkers L; Astuti GDN; Bennett J; Garanto A; van der Spuy J; Roepman R; Cheetham ME; Collin RWJ.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Medicaid coverage practices for approved gene and cell therapies: Existing barriers and proposed policy solutions.
Allen J; Berry D; Cook F; Hume A; Rouce R; Srirangam A; Wellman J; McCombs C.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Production and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard.
Mietzsch M; Liu W; Ma K; Bennett A; Nelson AR; Gliwa K; Chipman P; Fu X; Bechler S; McKenna R; Viner R.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
4/6 Transparent
Data Funding Declared
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-par…
Münter R; Christensen E; Andresen TL; Larsen JB.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis …
Kida S; Koshimura Y; Yoden E; Yoshioka A; Morimoto H; Imakiire A; Tanaka N; Tanaka S; Mori A; Ito J; Inoue A; Yamamoto R; Minami K; Hirato T; Takahashi K; Sonoda H.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Evaluation of diversity indices to estimate clonal dominance in gene therapy studies.
Corre G; Galy A.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
4/6 Transparent
Code Funding Declared
OCT4 induces long-lived dedifferentiated kidney progenitors poised to redifferentiate in 3D kidney spheroids.
Omer D; Zontag OC; Gnatek Y; Harari-Steinberg O; Pleniceanu O; Namestnikov M; Cohen AH; Nissim-Rafinia M; Tam G; Kalisky T; Meshorer E; Dekel B.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Process intensification for lentiviral vector manufacturing using tangential flow depth filtration.
Tona RM; Shah R; Middaugh K; Steve J; Marques J; Roszell BR; Jung C.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
An improved medium formulation for efficient <i>ex vivo</i> gene editing, expansion and engraftment of hematopoietic st…
Rai R; Naseem A; Vetharoy W; Steinberg Z; Thrasher AJ; Santilli G; Cavazza A.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Engineering olivocochlear inhibition to reduce acoustic trauma.
Zhang Y; Hiel H; Vincent PFY; Wood MB; Elgoyhen AB; Chien W; Lauer A; Fuchs PA.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Packaging cells for lentiviral vectors generated using the cumate and coumermycin gene induction systems and nanowell s…
Broussau S; Lytvyn V; Simoneau M; Guilbault C; Leclerc M; Nazemi-Moghaddam N; Coulombe N; Elahi SM; McComb S; Gilbert R.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Enrichment of transgene integrations by transient CRISPR activation of a silent reporter gene.
Mikkelsen NS; Hernandez SS; Jensen TI; Schneller JL; Bak RO.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Intravitreal air tamponade after AAV2 subretinal injection modifies retinal EGFP distribution.
Ducloyer JB; Pichard V; Mevel M; Galy A; Lefevre GM; Brument N; Alvarez-Dorta D; Deniaud D; Mendes-Madeira A; Libeau L; Le Guiner C; Cronin T; Adjali O; Weber M; Le Meur G.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Production of rAAV by plasmid transfection induces antiviral and inflammatory responses in suspension HEK293 cells.
Chung CH; Murphy CM; Wingate VP; Pavlicek JW; Nakashima R; Wei W; McCarty D; Rabinowitz J; Barton E.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
4/6 Transparent
Code Funding Declared