Gene therapy aims to introduce genetic information into a cell-type of interest to replace,correct,silence,or modify defective genes.Gene therapy in its broadest sense can theoretically prevent,halt,or cure any condit...Gene therapy aims to introduce genetic information into a cell-type of interest to replace,correct,silence,or modify defective genes.Gene therapy in its broadest sense can theoretically prevent,halt,or cure any condition that affects mankind.In addition to that,the introduction and/or manipulation of genes is one of the major research areas in biological sciences,aimed to deepen our knowledge on how biological systems work.Scientific advances have made it possible to induce changes ranging from manipulations of large stretches of the genome to the change of single nucleotides.The gold-standard vehicles to bring this genetic information into the target cells are viral vectors.展开更多
基金supported by the Royal Physiographic Society of Lund(Kungliga Fysiografiska sallskapet)the royal Swedish academy of sciences(Kungliga Vetenskapsakademien)+4 种基金the Per-Eric och Ulla Schybergs foundation(to MD)the Swedish Research Council(Vetenskapsradet)the Parkinsons foundation(Parkinsonsfonden)the Ahlens foundation(Ahlensstiftelsen)the Jeanssons foundation(Jeanssons stiftelser)as well as the Crafoord foundation(Crafoordska stiftelsen)(to AH)。
文摘Gene therapy aims to introduce genetic information into a cell-type of interest to replace,correct,silence,or modify defective genes.Gene therapy in its broadest sense can theoretically prevent,halt,or cure any condition that affects mankind.In addition to that,the introduction and/or manipulation of genes is one of the major research areas in biological sciences,aimed to deepen our knowledge on how biological systems work.Scientific advances have made it possible to induce changes ranging from manipulations of large stretches of the genome to the change of single nucleotides.The gold-standard vehicles to bring this genetic information into the target cells are viral vectors.