A 25-year-old woman with a history of acute lymphoblastic leukemia with Philadelphia chromosome (BCR 22q11.2-ABL1 (9q34)) presented with progressively worsening swelling and pain in her left upper eyelid of 3 months' duration. Magnetic resonance imaging showed a heterogeneously enhancing mass lesion in the left lacrimal gland.
View Article and Find Full Text PDFCRISPR (clustered regularly interspaced short palindromic repeats)/Cas gene editing is a revolutionary technology that can enable the correction of genetic mutations in vivo, providing great promise as a therapeutic intervention for inherited diseases. Adeno-associated viral (AAV) vectors are a potential vehicle for delivering CRISPR/Cas. However, they are restricted by their limited packaging capacity.
View Article and Find Full Text PDFAdeno-associated virus (AAV) vectors have been widely adopted for delivery of CRISPR-Cas components, especially for therapeutic gene editing. For a single vector system, both the Cas9 and guide RNA (gRNA) are encoded within a single transgene, usually from separate promoters. Careful design of this bi-cistronic construct is required due to the minimal packaging capacity of AAV.
View Article and Find Full Text PDFPurpose: Choroideremia is an X-linked retinal disease characterized by early retinal pigment epithelium (RPE) loss and subsequent retinal degeneration. The RPE adopts either a smooth or mottled appearance on fundus autofluorescence (FAF). It is not known how RPE changes predict the health of the overlying ellipsoid zone (EZ).
View Article and Find Full Text PDFPurpose: The Collaborative Ocular Melanoma Study (COMS) established iodine-125 (I-125) plaque brachytherapy for eye preserving treatment of medium-sized choroidal melanomas in the United States. Eye Physics I-125 plaque treatment modeled with Plaque Simulator (PS) software yields similar results to COMS. Herein, we report results from a series of 15 patients treated with ruthenium-106 (Ru-106) plaque brachytherapy using PS pretreatment modeling for plaque localization and dosimetry.
View Article and Find Full Text PDFImportance: The University of Southern California Eye Physics plaques compare favorably with the Collaborative Ocular Melanoma Study plaques in terms of late adverse effects from radiation, metastasis, and local tumor recurrence.
Objective: To review the University of Southern California experience using Eye Physics plaques and Plaque Simulator software to treat choroidal melanomas and compare the outcomes with published results of the Collaborative Ocular Melanoma Study.
Design, Setting, And Participants: A retrospective case series of 82 patients treated for medium-sized choroidal melanoma from January 1, 1990, through December 30, 2010, using iodine 125 plaques and treatment simulation software developed at the University of Southern California.