Results 1 to 10 of about 5,948,874 (305)

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

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

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

Quantification of Ocular Gene Therapy-Related Inflammation Using the VRMACS Grading System. [PDF]

Ophthalmol Sci
Abdalla Elsayed MEA, MacLaren RE.
europepmc   +1 more source

<i>In vivo</i> scAAV-based NeuroD1 gene therapy inhibiting glioblastoma growth. [PDF]

Mol Ther Oncol
Mazzetti D, Peruzzi PP, Soni H.
europepmc   +1 more source

Design and development of lipid nanoparticle formulations for brain gene therapy. [PDF]

Mol Ther Nucleic Acids
Thomson SB, de Moura Gomes A, Kazemian P, Kurek DZ, Kulkarni JA, Sadaati F, Wagner PK, Petkau TL, Ciufolini MA, Cullis PR, Leavitt BR.   +10 more
europepmc   +1 more source

Optimization of AAV tools to target Müller glial cells for retinal gene therapy. [PDF]

Mol Ther Adv
Urrutia-Cabrera D, Huppert G, Chu SWL, Wang L, Nguy CB, Liu CF, Lisowski L, Luu CD, Wang JH, Hung S, Hewitt AW, Huang CL, Edwards T, Martin KR, Wong RCB.   +14 more
europepmc   +1 more source

Overview of Patient-Reported Outcomes in Haemophilia Gene Therapy. [PDF]

Transfus Med Hemother
Miesbach W.
europepmc   +1 more source

Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies. [PDF]

Br J Haematol
Joshi G, Natarajan N, Dickerson KE, Voit RA.   +3 more
europepmc   +1 more source

P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up*

Genetics in Medicine Open
Francesca Fumagalli, Valeria Calbi, Fabiola De Mattia, Alberto Zambon, Vera Gallo, Salvatore Recupero, Elena Sophia Fratini, Alessia Ippolito, Francesca Ciotti, Maddalena Fraschini, Marina Sarzana, Stefano Scarparo, Eugenio Montini, Sara Locatelli, Marcella Facchini, Alessandra Clerici, Francesco Morena, Sabata Martino, Muska Yarzi, Sean Moro, Andrew Shenker, Jean Brooks, Alan Richardson, Leslie Meltzer, Laura Campbell, Alessandro Aiuti   +25 more
doaj   +1 more source

New Gene Therapy Strategy for β-Thalassemia. [PDF]

Stem Cell Rev Rep
Liang D, Schmidt-Wolf IGH, Pu J.
europepmc   +1 more source