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We narrowed to 67 results for: rpe

Showing: 1 - 20 of 67 results

  1. AAVs in Retinal Gene Therapy

    Type
    Blog Post
    ...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...

  3. Using AAV for Neuronal Tracing

    Type
    Blog Post
    ...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...

  4. X-CHIME: Context Dependent Germline Knockout in Immune Cells

    Type
    Blog Post
    ..., 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...

  5. Celebrating Outstanding CRISPR/Cas9 Achievements at the Dr. Paul Janssen Award Dinner

    Type
    Blog Post
    ...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...

  6. Antibodies 101: Flow Compensation

    Type
    Blog Post
    ...against each other, you should be able to draw perpendicular lines that intersect your populations. If you...population needs to be shifted, consider your perpendicular lines. In this case, the population on the left...needs to shift towards the X axis to become a perpendicular vertical line.     In general, most spillover...She is currently a research technician in Arlene Sharpe's lab at Harvard Medical School.   More resources...

  7. Performing In Vivo CRISPR Screens Using the FITS Approach

    Type
    Blog Post
    ..., developed and deposited at Addgene by Arlene Sharpe’s lab, enables in vivo screens in naïve T cells....Marty LaFleur is a Postdoctoral Fellow in Arlene Sharpe’s laboratory at Harvard Medical School and is interested..., Hacohen, N., Doench, J. G., LaFleur, M. W., & Sharpe, A. H. (2024). Framework for in vivo T cell screens...

  8. Adeno Associated Virus (AAV) for Cell and Gene Therapy

    Type
    Blog Post
    ... 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...

  9. Typing CRISPR Systems

    Type
    Blog Post
    ...science.adi1910 Chylinski, K., Makarova, K. S., Charpentier, E., & Koonin, E. V. (2014). Classification and...Saunders, S. J., Barrangou, R., Brouns, S. J. J., Charpentier, E., Haft, D. H., Horvath, P., Moineau, S., Mojica... S. A., Alkhnbashi, O. S., Brouns, S. J. J., Charpentier, E., Cheng, D., Haft, D. H., Horvath, P., Moineau...

  11. Light Sheet Fluorescence Microscopy

    Type
    Blog Post
    ...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...

  12. CRISPR 101: Making a Knock-In Cell Line

    Type
    Blog Post
    ...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...

  13. Plasmids 101: Repressible Promoters

    Type
    Blog Post
    ...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. ...

  15. Neurodegeneration Plasmid Collection

    Type
    Collection
    ...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...

  16. CRISPR History and Development for Genome Engineering

    Type
    Collection
    ... Chylinski K, Fonfara I, Hauer M, Doudna JA, Charpentier E. 2012. A programmable dual-RNA-guided DNA endonuclease...Shah SA, Saunders SJ, Barrangou R, Brouns SJ, Charpentier E, Haft DH, Horvath P, Moineau S, Mojica FJ, ...
Showing: 1 - 20 of 67 results