Surgical outcomes for patients with CNS hemangioblastomas are favorable. However, management of hemangioblastomas is a more difficult and prolonged endeavor for patients with VHL syndrome. In patients with VHL syndrome, neuroradiological screening allows identification of lesions before they become symptomatic. Because patients with VHL syndrome are at risk for development of new lesions, they require lifelong follow-up.
Recombinant adeno-associated virus type 2 (rAAV) vecderived cell line. Effective amplification of rAAV vectors tors have recently been used to achieve long-term, high introduced into 293 cells by infection was also demonlevel transduction in vivo. Further development of rAAV strated. Passage of rAAV with d27.1-rc results in up to 200-vectors for clinical use requires significant technological fold amplification of AAV-GFP with each passage after improvements in large-scale vector production. In order to coinfection of the vectors. Efficient, large-scale production facilitate the production of rAAV vectors, a recombinant (Ͼ10 9 cells) of AAV-GFP from a proviral cell line was also herpes simplex virus type I vector (rHSV-1) which does not achieved and these stocks were free of replication-comproduce ICP27, has been engineered to express the AAVpetent AAV. The described rHSV-1 vector provides a 2 rep and cap genes. The optimal dose of this vector, novel, simple and flexible way to introduce the AAV-2 rep d27.1-rc, for AAV production has been determined and and cap genes and helper virus functions required to proresults in a yield of 380 expression units (EU) of AAV-GFP duce high-titer rAAV preparations from any rAAV proviral produced from 293 cells following transfection with AAVconstruct. The efficiency and potential for scalable delivery GFP plasmid DNA. In addition, d27.1-rc was also efficient of d27.1-rc to producer cell cultures should facilitate the at producing rAAV from cell lines that have an integrated production of sufficient quantities of rAAV vectors for clini-AAV-GFP provirus. Up to 480 EU/cell of AAV-GFP could cal application. be produced from the cell line GFP-92, a proviral, 293
The endonasal and transoral approaches allow wide exposure with large working angles to the craniocervical junction. The transcervical approach accesses the odontoid for resection from the body of C2 to the lip of the basion. The angles of attack in the transcervical approach when centered on the surgical target are limited, but this approach offers a clean, sterile operative field. Clinical investigation will be required to determine the optimal indications for each approach.
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