Award Data

Project SummaryImpairment in instrumental activities of daily living (IADL) is a hallmark of Alzheimer’s disease (AD) dementia and a major source of patient and caregiver burden. Similarly to cognitive changes in AD, subtle difficulties in IADL may begin even before the stage of amnestic mild cognitive impairment (MCI). Recently, greater emphasis has been placed on preclinical AD, which consists ...

PROJECT SUMMARY/ABSTRACT Post-traumatic osteoarthritis (PTOA) is a degenerative disease of cartilage brought on by traumatic injury to the articular joints. Acute joint injury is followed by severe joint pain and inflammation. This results in much more rapid degeneration of cartilage than in other forms of osteoarthritis, due to the joint instability caused by trauma as well as the increase in pro ...

PROJECT SUMMARY Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic disorder in the world and affects 650,000 Americans. ADPKD is caused by mutations in Pkd1 or Pkd2 that trigger proliferation and fluid secretion by renal tubular epithelial cells into cysts, which bilaterally enlarge the kidney and lead to progressive loss of kidney function. There is no cure for ADPK ...

AbstractBony fracture is one of the most challenging musculoskeletal injuries in the elderly population. In clinic, a significant decline in bone healing potential, accompanying with mental and life- threatening complications, is commonly observed in elderly who are 65 years or older. With the rapid growth of elderly population in the US, it imposes a substantial cost burden on the health care sys ...

ABSTRACT Despite treatment advances, the prognosis of adult patients with relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL) and R/R diffuse large B-cell lymphoma (DLBCL) remains poor. Autologous T- cells modified to express a CD19-targeted chimeric antigen receptor (CAR T-cells) produce durable responses in subgroups of these patients, which has led to FDA approval of two su ...

1 ABSTRACT2 Tuberous sclerosis (TS), also called tuberous sclerosis complex, is a rare, multi-systemic genetic and often life-3 threatening disease that causes benign tumors to grow in the brain and on other vital organs such as the kidneys,4 heart, eyes, lungs, and skin. Lymphangioleiomyomatosis (LAM) is a TS-related tumor-like disorder. Both occur5 as a consequence of an inherited or sporadic mu ...

ABSTRACT Lymphangioleiomyomatosis (LAM) is a Tuberous sclerosis-related disorder. Both occur due to an inherited or sporadic mutation in either the TSC1 or TSC2 gene, which function as negative regulators of the mTOR pathway. Uncontrolled mTORC1 activity leads to the neoplastic proliferation of abnormal smooth muscle cells (LAM cells) in the lungs, progressive shortness of breath, recurrent pneumo ...

Abstract – sxRNA Technologies (sxRNATech) is developing an organoid toolkit for Alzheimer’s Disease (AD) and Alzheimer's-Disease-Related Dementias (ADRD) that will enable the identification of senescent cells in living tissue providing a novel tool for studying senescence and how it relates to the aging processes while also creating a new platform for high-throughput drug screening in in compl ...

ABSTRACT This STTR application proposes a new platform, the Clinical Picture Maker, which aims to facilitate the production, organization, and dissemination of video documentation that can improve the diagnosis and treatment of rare and difficult to diagnose disorders. At present, people with rare disorders and their caregivers often face a 5+ year search for a diagnosis, which can lead to serious ...

Food allergy (FA), a potentially life-threatening condition, has rapidly increased for 2 decades, affecting 32 million Americans with annual costs of $25 billion. Treatment options are extremely limited. Food avoidance and rescue medication after accidental exposure are primary to FA management. Peanut allergy (PNA) causes severe reactions, often co-existing with tree nut allergies (TNA). Shellfis ...