GKA deel 1 college 5

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy Cartier, Hacein-Bey-Abina, Von Kalle, Fischer, Cavazzana-Calvo and others Science (2009) 326: 818 – 823 ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],Inserted Nov 2009

Genetische therapiën ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

RNA interference; RNAi Pomerantz 2002, Nature Med 8, 659-660 ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Different small RNAs Großhans and Filipowicz, Nature 451, 2008, 414-416 ,[object Object]

Can RNAi be a therapy? Stevenson, Nature Rev Imm 3, 2003, 851-858 ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],Viral (m)RNA

HIV life cycle Stevenson, Nature Rev Imm 3, 2003, 851-858

siRNA inhibits HIV infection ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

How to make RNA? Stevenson, Nature Rev Imm 3, 2003, 851-858 One promoter

How to make RNA? Stevenson, Nature Rev Imm 3, 2003, 851-858 One promoter Two promoters

How to make RNA? Stevenson, Nature Rev Imm 3, 2003, 851-858 One promoter Two promoters Viral vector: adeno, HIV

Factors influencing effect of RNAi Stevenson, Nature Rev Imm 3, 2003, 851-858 ,[object Object],[object Object],[object Object],[object Object]

Factors influencing effect of RNAi Stevenson, Nature Rev Imm 3, 2003, 851-858 ,[object Object],[object Object],[object Object],[object Object],[object Object]

Delivery to brain cells Nature 448, 2007, 33-34 ,[object Object],Kumar et al ., Nature 448, 2007, 39-43, Transvascular delivery of small interfering RNA to the central nervous system Inserted July 10, 2007 ========

GKA deel 1 college 5

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