Intravitreal Faricimab for Previously Treated Neovascular Age-Related Macular Degeneration.

Purpose: To report our real-world experience using intravitreal faricimab, a novel anti-vascular endothelial growth factor (anti-VEGF) therapy, in eyes with neovascular age-related macular degeneration (nAMD) previously treated with other anti-VEGF therapy.

Patients And Methods: A retrospective, single-center study of previously treated nAMD eyes treated with faricimab.

Results: In 88 eyes (73 patients), mean baseline best-corrected visual acuity (BCVA) was 20/63 (range 20/20 to CF) with mean anti-VEGF injection interval of 6.

Imaging Phospholipase D Activity with Clickable Alcohols via Transphosphatidylation.

Phospholipase D (PLD) is an enzyme with many functions, one of which is the synthesis of phosphatidic acid (PA), a molecule with a myriad of effects on various organ systems and processes. These numerous roles make it hard to understand the true action of PA in cellular and bodily processes. Imaging PLD activity is one way to better understand the synthesis of PA and start to elucidate its function.

Cryptogenic Pontine Abscess Treated With Stereotactic Aspiration: A Case Report.

Brainstem abscesses are localized collections of pus or infected material within the brainstem, which can cause inflammation, tissue damage, and compression of adjacent structures. This can lead to a variety of symptoms, including headache, fever, and focal neurological deficits, among many others. Brainstem abscesses are potentially life-threatening and considered to be rare, and pontine abscesses are even rarer.

Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial.

Background: Primary hyperoxaluria type 1 (PH1) is a rare genetic disease that causes progressive kidney damage and systemic oxalosis due to hepatic overproduction of oxalate. Lumasiran demonstrated efficacy and safety in the 6-month primary analysis period of the phase 3, multinational, open-label, single-arm ILLUMINATE-B study of infants and children < 6 years old with PH1 (ClinicalTrials.gov: NCT03905694 (4/1/2019); EudraCT: 2018-004,014-17 (10/12/2018)).

CRISPR-based VEGF suppression using paired guide RNAs for treatment of choroidal neovascularization.

Clustered regularly interspaced short palindromic repeats (CRISPR)-based genomic disruption of vascular endothelial growth factor A () with a single gRNA suppresses choroidal neovascularization (CNV) in preclinical studies, offering the prospect of long-term anti-angiogenesis therapy for neovascular age-related macular degeneration (AMD). Genome editing using CRISPR-CRISPR-associated endonucleases (Cas9) with multiple guide RNAs (gRNAs) can enhance gene-ablation efficacy by augmenting insertion-deletion (indel) mutations with gene truncations but may also increase the risk of off-target effects. In this study, we compare the effectiveness of adeno-associated virus (AAV)-mediated CRISPR-Cas9 systems using single versus paired gRNAs to target two different loci in the gene that are conserved in human, rhesus macaque, and mouse.

Randomized Clinical Trial on the Long-Term Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1.

Introduction: Primary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by hepatic overproduction of oxalate, leading to kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. In the 6-month double-blind period (DBP) of ILLUMINATE-A, a phase 3, randomized, placebo-controlled trial in patients with PH1 ≥6 years old, treatment with lumasiran, an RNA interference therapeutic, led to substantial reductions in urinary oxalate (UOx) levels.

Methods: We report data to month 12 in the extension period (EP) of ILLUMINATE-A, including patients who continued lumasiran (lumasiran/lumasiran) or crossed over from placebo to lumasiran (placebo/lumasiran).

CRISPR Technology for Ocular Angiogenesis.

Among genome engineering tools, Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-based approaches have been widely adopted for translational studies due to their robustness, precision, and ease of use. When delivered to diseased tissues with a viral vector such as adeno-associated virus, direct genome editing can be efficiently achieved to treat different ophthalmic conditions. While CRISPR has been actively explored as a strategy for treating inherited retinal diseases, with the first human trial recently initiated, its applications for complex, multifactorial conditions such as ocular angiogenesis has been relatively limited.

Host Immune Responses after Suprachoroidal Delivery of AAV8 in Nonhuman Primate Eyes.

The suprachoroid is a potential space located between the sclera and choroid of the eye, which provides a novel route for ocular drug or viral vector delivery. Suprachoroidal injection of adeno-associated virus (AAV)8 using transscleral microneedles enables widespread transgene expression in eyes of nonhuman primates, but may cause intraocular inflammation. We characterized the host humoral and cellular immune responses after suprachoroidal delivery of AAV8 expressing green fluorescent protein (GFP) in rhesus macaques, and found that it can induce mild chorioretinitis that resolves after systemic corticosteroid administration, with recovery of photoreceptor morphology, but persistent immune cell infiltration after 3 months, corresponding to a loss of GFP expression from retinal pigment epithelial cells, but persistent expression in scleral fibroblasts.

Race/Ethnicity, Primary Language, and Income Are Not Demographic Drivers of Mortality in Breast Cancer Patients at a Diverse Safety Net Academic Medical Center.

Objective. To examine the impact of patient demographics on mortality in breast cancer patients receiving care at a safety net academic medical center. Patients and Methods.

Patient demographic characteristics and disease stage as drivers of disparities in mortality in prostate cancer patients who receive care at a safety net academic medical center.

Introduction: The purpose of this study was to examine the effect of patient demographic characteristics and tumor stage at diagnosis on mortality in prostate cancer patients who receive care at a safety net, academic medical center with a diverse patient population.

Patients And Methods: Eight hundred sixty-nine patients were diagnosed with prostate cancer at our institution between August 2004 and October 2011. Patient demographic characteristics were determined as follows: race and/or ethnicity, primary language, insurance type, age at diagnosis, marital status, income (determined by zip code), and American Joint Committee on Cancer (AJCC) tumor stage.

Circulating tumor cells as predictors of response and failure in breast cancer patients treated with preoperative chemotherapy.

Aim: To explore the significance of circulating tumor cells (CTCs) detection in the course of preoperative chemotherapy (PC) and their effect on the outcomes.


Methods: Fifty-five patients with stage II/III invasive breast cancer were enrolled into a preoperative clinical trial. Patients were given PC with sequential single-agent doxorubicin and paclitaxel vs paclitaxel followed by doxorubicin.