Results 81 to 90 of about 2,056 (145)

Chemically modified AAV for gene therapy

, 2021
Du fait de leurs propriétés, les virus adéno-associés ou AAV, sont devenus des vecteurs de choix pour le transfert de gênes thérapeutiques. Leur efficacité a été largement démontrée et ils sont aujourd’hui utilisés dans plus de cent essais cliniques. Ils
Leray, Aurelien
core  

Regulating synthetic biology and advanced genetic interventions: biosecurity frameworks, gene therapy oversight, stem-cell manufacturing risks and future policy directions

Journal of Biosafety and Biosecurity
Gene editing (CRISPR-Cas9, base and prime editing), viral-vector therapies (adeno-associated virus [AAV] and lentiviral systems) and engineered stem cell platforms (induced pluripotent stem cells and mesenchymal lines) have transformed disease treatment ...
Neeraja Suresh, Anushka Rathore, Vijay Singh, Shailendra Dwivedi, Akash Bansal   +4 more
doaj   +1 more source

Θεραπευτικές προσεγγίσεις στη συγγενή αμαύρωση Leber

, 2023
Η συγγενής αμαύρωση Leber (LCA) είναι μια ομάδα κληρονομικών ασθενειών του αμφιβληστροειδούς που χαρακτηρίζονται από σοβαρή εξασθένηση της όρασης ή τύφλωση κατά τη γέννηση. Η LCA είναι μια σοβαρή μορφή μελαγχρωστικής αμφιβληστροειδοπάθειας (RP). Η πάθηση
Ταπανλής Άγγελος, Tapanlis Angelos   +1 more
core  

Clinical applications of gene therapy for rare diseases: A review [PDF]

, 2023
Rare diseases collectively exact a high toll on society due to their sheer number and overall prevalence. Their heterogeneity, diversity, and nature pose daunting clinical challenges for both management and treatment.
Papaioannou, Ioannis, Owen, James S, Yáñez-Muñoz, Rafael J   +2 more
core  

Affinity Capture Approaches for AAV Purification

, 2022
Currently, there has been a period marked by developments in the field of gene therapy, focused on personalized medicine. Therapies based on direct and targeted in vivo delivery of the gene load, such as Glybera, Luxturna or Zolgensma, stand out as the ...
Santos, Miguel Barbosa Louro dos
core  

Precision Medicine in Inherited Retinal Disease: Advances, Challenges, and Future Directions. [PDF]

J Pers Med
Dhivagaran T, Butt FR, Grover K, Devgan K, Sachdeva K, Dhivagaran V, Abid F, Tao BK, Balas M, Dimopoulos I, Bhatti A.   +10 more
europepmc   +1 more source

Multi-Luminance Mobility Test (MLMT) as a tool for assessing functional vision in patients with RPE65 gene mutations following gene therapy (voretigene neparvovec)

Klinika Oczna
Martyna Mieszczak, Marika Pitnoczko, Kinga Obst, Zuzanna Niedziela-Schwartz, Piotr Rakowicz, Maciej Krawczyński, Marcin Stopa   +6 more
doaj   +1 more source

Small populations, big impact: leveraging rare disease gene therapies to benefit millions. [PDF]

J Clin Invest
Cherqui S.
europepmc   +1 more source

Clinical and pharmacovigilance safety evaluation of LUXTURNA^® (voretigene neparvovec-rzyl). [PDF]

Cutan Ocul Toxicol
Simoens D, Shravah V, Jones WK, Kaja S.
europepmc   +1 more source

One down but many more to go: the state of gene therapy for inherited retinal disease. [PDF]

Regen Med
Tan TE, Sun CZY, Poh SSJ, Lim J, Teo JXJ, Dey S, Chua ZWX, Guo J, Wang Z, Tay HG, Fenner BJ.   +10 more
europepmc   +1 more source