Research Papers

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CRISPR-Cas9-based non-viral gene editing therapy for topical treatment of recessive dystrophic epidermolysis bullosa.
Wang X; Wang X; Li Y; A S; Qiu B; Bushmalyova A; He Z; Wang W; Lara-Sáez I.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Mitochondrial transplantation reduces lower limb ischemia-reperfusion injury by increasing skeletal muscle energy and a…
Zeng J; Liu J; Ni H; Zhang L; Wang J; Li Y; Jiang W; Wu Z; Zhou M.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Molecular and functional correction of a deep intronic splicing mutation in <i>CFTR</i> by CRISPR-Cas9 gene editing.
Walker AJ; Graham C; Greenwood M; Woodall M; Maeshima R; O'Hara-Wright M; Sanz DJ; Guerrini I; Aldossary AM; O'Callaghan C; Baines DL; Harrison PT; Hart SL.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
4/6 Transparent
Data Funding Declared
<i>A novel FOXP3</i> knockout-humanized mouse model for pre-clinical safety and efficacy evaluation of Treg-like cell p…
Sato Y; Nathan A; Shipp S; Wright JF; Tate KM; Wani P; Roncarolo MG; Bacchetta R.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Multiparametric domain insertional profiling of adeno-associated virus VP1.
Hoffmann MD; Zdechlik AC; He Y; Nedrud D; Aslanidi G; Gordon W; Schmidt D.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
5/6 Transparent
Data Code Funding Declared
Enhancement of recombinant adeno-associated virus activity by improved stoichiometry and homogeneity of capsid protein …
Onishi T; Nonaka M; Maruno T; Yamaguchi Y; Fukuhara M; Torisu T; Maeda M; Abbatiello S; Haris A; Richardson K; Giles K; Preece S; Yamano-Adachi N; Omasa T; Uchiyama S.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model.
Benatti HR; Prestigiacomo RD; Taghian T; Miller R; King R; Gounis MJ; Celik U; Bertrand S; Tuominen S; Bierfeldt L; Parsley E; Gallagher J; Hall EF; McElroy AW; Sena-Esteves M; Khvorova A; Aronin N; Gray-Edwards HL.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy.
Ellison S; Liao A; Gleitz HFE; Parker H; Booth L; Robinson J; Wood S; Taylor J; Holley R; Bigger BW.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Characterization and AAV-mediated <i>CRB</i> gene augmentation in human-derived <i>CRB1</i><sup>KO</sup> and <i>CRB1</i…
Boon N; Lu X; Andriessen CA; Orlovà M; Quinn PMJ; Boon CJF; Wijnholds J.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Clinical holds for cell and gene therapy trials: Risks, impact, and lessons learned.
Wills CA; Drago D; Pietrusko RG.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
2/6 Transparent
Optogenetic targeting of AII amacrine cells restores retinal computations performed by the inner retina.
Khabou H; Orendorff E; Trapani F; Rucli M; Desrosiers M; Yger P; Dalkara D; Marre O.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
5/6 Transparent
Data Code Funding Declared
Inhibition of DNA-dependent protein kinase catalytic subunit boosts rAAV transduction of polarized human airway epithel…
Ning K; Zhang X; Feng Z; Hao S; Kuz CA; Cheng F; Park SY; McFarlin S; Engelhardt JF; Yan Z; Qiu J.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Mini-TCRs: Truncated T cell receptors to generate T cells from induced pluripotent stem cells.
Takayanagi SI; Wang B; Hasegawa S; Nishikawa S; Fukumoto K; Nakano K; Chuganji S; Kato Y; Kamibayashi S; Minagawa A; Kunisato A; Nozawa H; Kaneko S.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Production of therapeutic levels of human FIX-R338L by engineered B cells using GMP-compatible medium.
David M; Monteferrario D; Saviane G; Jeanneau C; Marchetti I; Dupont CF; Dumont C; Fontenot JD; Rosa M; Fenard D.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
2/6 Transparent
Development of an automated manufacturing process for large-scale production of autologous T cell therapies.
Francis N; Braun M; Neagle S; Peiffer S; Bohn A; Rosenthal A; Olbrich T; Lollies S; Ilsmann K; Hauck C; Gerstmayer B; Weber S; Kirkpatrick A.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
2/6 Transparent
Oxidative stress induced by sustained supraphysiological intrastriatal GDNF delivery is prevented by dose regulation.
Azevedo MD; Prince N; Humbert-Claude M; Mesa-Infante V; Jeanneret C; Golzne V; De Matos K; Jamot BB; Magara F; Gonzalez-Hernandez T; Tenenbaum L.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
4/6 Transparent
Data Funding Declared
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy.
Le Guiner C; Xiao X; Larcher T; Lafoux A; Huchet C; Toumaniantz G; Adjali O; Anegon I; Remy S; Grieger J; Li J; Farrokhi V; Neubert H; Owens J; McIntyre M; Moullier P; Samulski RJ.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Correlating physicochemical and biological properties to define critical quality attributes of a rAAV vaccine candidate.
Kumar P; Wang M; Kumru OS; Hickey JM; Sanmiguel J; Zabaleta N; Vandenberghe LH; Joshi SB; Volkin DB.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Mechanistic modeling explains the production dynamics of recombinant adeno-associated virus with the baculovirus expres…
Destro F; Joseph J; Srinivasan P; Kanter JM; Neufeld C; Wolfrum JM; Barone PW; Springs SL; Sinskey AJ; Cecchini S; Kotin RM; Braatz RD.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
4/6 Transparent
Code Funding Declared
In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV ser…
Schwartz MK; Likhite S; Vetter TA; Baird MC; McGovern V; Sierra Delgado A; Mendel T; Burghes A; Meyer KC.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined t…
Lisjak M; Iaconcig A; Guarnaccia C; Vicidomini A; Moretti L; Collaud F; Ronzitti G; Zentilin L; Muro AF.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Cytokine and reactivity profiles in SLE patients following anti-CD19 CART therapy.
Nunez D; Patel D; Volkov J; Wong S; Vorndran Z; Müller F; Aigner M; Völkl S; Mackensen A; Schett G; Basu S.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
2/6 Transparent
CRISPR-Cas9 homology-independent targeted integration of exons 1-19 restores full-length dystrophin in mice.
Stephenson AA; Nicolau S; Vetter TA; Dufresne GP; Frair EC; Sarff JE; Wheeler GL; Kelly BJ; White P; Flanigan KM.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared
Development of an <i>in vitro</i> genotoxicity assay to detect retroviral vector-induced lymphoid insertional mutants.
Bastone AL; Dziadek V; John-Neek P; Mansel F; Fleischauer J; Agyeman-Duah E; Schaudien D; Dittrich-Breiholz O; Schwarzer A; Schambach A; Rothe M.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
4/6 Transparent
Data Funding Declared
Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes.
Kok CY; Tsurusaki S; Cabanes-Creus M; Igoor S; Rao R; Skelton R; Liao SHY; Ginn SL; Knight M; Scott S; Mietzsch M; Fitzsimmons R; Miller J; Mohamed TMA; McKenna R; Chong JJH; Hill AP; Hudson JE; Alexander IE; Lisowski L; Kizana E.
Mol Ther Methods Clin Dev (Unknown)
Published: 2023
3/6 Transparent
Funding Declared