...infecting cells of the retinal pigmented epithelium (RPE). AAVs, on the other hand, have broad tropism in...Luckily for patients, this gene can be targeted to RPE cells using a lentiviral vector and a clinical trial...after the cloning of the RPE65 gene, a naturally occurring animal model, the RPE65−/− dog, was characterized...of several disease-causing genes, including the RPE65 gene responsible for an autosomal recessive, severe...study showing that AAV-mediated delivery of the RPE65 gene restores vision in the dog model of LCA (Lancelot...Lancelot, one of the dogs who received the AAV-RPE65 treatment, famously visited Congress to help increase...

...Pancreas AAV8 Photoreceptor Cells AAV2, AAV5, AAV8 RPE (Retinal Pigment Epithelium) AAV1, AAV2, AAV4, AAV5...

...AAV7, AAV8, AAV9 Lung AAV4, AAV5, AAV6, AAV9 RPE (Retinal Pigment Epithelium) AAV1, AAV2, AAV4, AAV5...untreated (for review see Dietzschold et al., 2008). Herpes Simplex Virus (HSV) also migrates through neurons... Strick, P.L. (1989). Transneuronal transfer of herpes virus from peripheral nerves to cortex and brainstem...

...Pancreas AAV8 Photoreceptor Cells AAV2, AAV5, AAV8 RPE (Retinal Pigment Epithelium) AAV1, AAV2, AAV4, AAV5...without help. Another virus, such as adenovirus or herpes simplex virus, or a genotoxic agent such as UV ...

..., developed and deposited at Addgene by Arlene Sharpe’s lab, allow for wide deployment of in vivo gene...Marty LaFleur is a Postdoctoral Fellow in Arlene Sharpe’s laboratory at Harvard Medical School and is interested...Montero Llopis, P., Haining, W. N., Doench, J. G., & Sharpe, A. H. (2024). X-CHIME enables combinatorial, inducible..., Al Abosy, R., Freeman, G.J., Haining, W.N., & Sharpe, A.H. (2019). PTPN2 regulates the generation of..., Coxe, D. J., Doench, J. G., Haining, W. N., & Sharpe, A. H. (2019). A CRISPR-Cas9 delivery system for...

...engineering, Dr. Jennifer Doudna and Dr. Emmanuelle Charpentier. I was joined by Addgene Scientist Matt Ferenc...and Joan Steitz. This award ceremony honored Charpentier and Doudna, who talked about their research leading...simpler 1 protein - 1 RNA system Drs. Dounda and Charpentier met at a 2011 conference and decided to collaborate...was a simpler 1 protein - 1 RNA system.  Dr. Charpentier advocated for the crucial role of basic research...

... the human gene RPE65, patients with clinical diagnosis of LCA and mutations in RPE65 (n=3), showed significant...This disease is caused by mutations in the gene RPE65. It is characterized by childhood onset of blindness...

...especially race and gender) Interpersonal issues can create interpersonal issues (one bad apple can change...

...light using a different objective lens oriented perpendicular to the plane of illumination (i.e. light sheet...Klymkowsky. (1989) Development, 105.61-74. Pubmed. J. Sharpe, U. Ahlgren, P. Perry, Hill B, Ross A, J. Hecksher-Sorensen...

...potentially shuttling repair down a different pathway (Charpentier et al., Nat Com).     Fig. 3: HDR events... 8(2024), (2017). 10.1038/s41467-017-01836-2 Charpentier, M., Khedher, A. H. Y., Menerot, S., Brion, A...

...transcriptional activation domain VP16 taken from the herpes simplex virus. tTA binds to promoters containing...Functional dissection of VP16, the trans-activator of herpes simplex virus immediate early gene expression. ...

...Using acrylamide instead of agarose gives much sharper bands. I compared two MOIs of gRNA transduction... fragments, run PAGE instead of agarose to get sharper bands. Conclusion Overall, making a human cell...

...Parkinson's Wade Harper 22594 Flag-HA-OTUD4 OTUD4 HA, FLAG CMV Gordon Holmes Syndrome Wade Harper 23026 pET9d-hnRNP-A1...pHAGE-eGFP-OPTN OPTN ALS Wade Harper 175760 pHAGE-FLAG-APEX2-OPTN OPTN Flag CMV ALS Wade Harper 175762 pHAGE-eGFP-...GFP CMV ALS Wade Harper 175763 pHAGE-eGFP-OPTN S473A 513A OPTN GFP CMV ALS Wade Harper 175764 pHAGE-eGFP-OPTN...OPTN GFP CMV ALS Wade Harper 175765 pHAGE-eGFP-OPTN E50K OPTN GFP CMV ALS Wade Harper 176267 pAAV-FLEX-P301L...177094 pHAGE-APPSw;T719N APP CMV Alzheimer's Wade Harper 177511 Anti-Frataxin [N191/7R] FXN CMV Friedreich...Cookson 193772 pENTR221-APP751 APP Alzheimer's Wade Harper 194004 VPS13D(deltaATG2-C/PH)^EGFP VPS13D GFP CMV...

...through obstacles, and take steps to resolve interpersonal conflicts in the lab. Yearly planning meetings...

...that you see in person, and helps improve your interpersonal relationships with people you’re collaborating...

...Makarova K, Haft DH, Barrangou R, Brouns SJJ, Charpentier E, Horvath P, Moineu S, Mojica FJM, Wolf YI, ...

...tagging?   References and Resources References Terpe, K., Overview of tag protein fusions: from molecular...

...learning. Storytelling is a crucial part of interpersonal communication, which makes honing your ability...

...Charlotte, all of whom are still working on theirpenmanship. Additional resources on the Addgene blog ...

...are no "natural" mammalian ORIs, scientists have usurped viral-based ORIs to fill the void. These ORIs, ...