Results 231 to 240 of about 6,300,245 (266)

Quantitative Dose Optimization for Gene Therapy Trials: Implications for Project Optimus and Early-Phase Design. [PDF]

Pharm Stat
Roberts K, Palmer J, Volpe J, Zhong W, McIntosh A.   +4 more
europepmc   +1 more source

Delivery of DNA‐encoded vaccines and proteins in mice using cutaneous suction‐mediated transfection

FEBS Open Bio, EarlyView.
Cutaneous suction‐mediated transfection in mice for delivery of DNA‐encoded vaccines and proteins. A critical limitation of DNA vaccines and other therapeutics is transfection in vivo to produce the encoded antigens or therapeutic proteins. Cutaneous suction‐based methods have demonstrated effectiveness in many animal models and have been successfully ...
Emran O. Lallow, Isabel Brandtjen, Yaxin Mo, Melissa Gulley, Louis Osorio, Sagar Kudchodkar, Nandita C. Jhumur, Christine C. Roberts, Lisa K. Denzin, David I. Shreiber, Biju Parekkadan, Hao Lin, Joel N. Maslow   +12 more
wiley   +1 more source

Biodegradable Acoustic Targeting for Ultrasound-Supported Gene Therapy (BATUS) in Glioblastoma. [PDF]

Adv Sci (Weinh)
Erel-Akbaba G, Park J, Duhoon A, Nguyen CTG, Bhaskar N, Karan SK, Akbaba H, Singh P, Tran TBT, Zhu Y, Li Z, Muhammad I, Zhang X, Truong HQ, Davidar AD, Schmitzer E, Vattipally VN, Lopez AF, Kramer P, Quiroz VM, Bykov B, Hao C, Manbachi A, Theodore N, Doloff JC, Nguyen TD.   +25 more
europepmc   +1 more source

Therapeutic Window of AAV-SIRT1 Gene Therapy in Mouse Models of Optic Nerve Degeneration. [PDF]

Transl Vis Sci Technol
Chaqour B, Pham JC, Yue J, Dine K, Jin F, Ying GS, Bennett J, Shindler KS, Ross AG.   +8 more
europepmc   +1 more source

Gene therapy for hereditary hematological disorders: From clinical breakthroughs to future horizons. [PDF]

Mol Ther Nucleic Acids
Chen Z, Wang W, Wu Y, Qiang Y, Cheng W, Liu F.   +5 more
europepmc   +1 more source

BAG3 gene therapy emerges as a potential treatment for myofibrillar myopathies. [PDF]

Mol Ther Adv
Sherer LA, Kirk JA.
europepmc   +1 more source

AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich Ataxia: A Nonrandomized Clinical Trial.

JAMA Cardiol
Crystal RG, Weinsaft JW, Kaminsky SM, Caragiulo A, Savage N, Patel A, Gavrilova RH, Perlman SL, Galbraith M, Kahlon U, Krishnan U, Kaner RJ, Sanders A, Vo M, Sarva H, Yoo A, Sondhi D, De BP, Mezey JG, Aubert G, Khan A, Selvan N, Bhalla N, Adler E, Zesiewicz T.   +24 more
europepmc   +1 more source