Results 201 to 210 of about 287,515 (303)

Modulation of the Stress Granule Component Carhsp1 Mitigates Disease‐Associated Deficits in Spinocerebellar Ataxia Type 3 Mouse Models

Movement Disorders, EarlyView.
Abstract Background Spinocerebellar ataxia type 3 (SCA3) is a polyglutamine (polyQ) neurogenerative disorder that results from CAG trinucleotide repeat expansions in the ATXN3 gene, leading to toxic protein aggregate formation and cellular pathway dysfunction.
Tiago Moreira‐Gomes, Adriana Marcelo, Ana Teresa Rajado, Rafael G. Costa, Ricardo Afonso‐Reis, Cristiana R. Madeira, Inês T. Afonso, David V.C. Brito, Adriana A. Vaz, Clévio Nóbrega   +9 more
wiley   +1 more source

Correction: Diversity of transgene integration and gene-editing events in wheat (<i>Triticum aestivum</i> L.) transgenic plants generated using <i>Agrobacterium</i>-mediated transformation. [PDF]

Front Genome Ed
Lopos LC, Bykova NV, Robinson J, Brown S, Ward K, Bilichak A.   +5 more
europepmc   +1 more source

An inducible caspase 9 safety switch for T-cell therapy [PDF]

, 2005
Brenner, MK, Dotti, G, Heslop, HE, Pule, MA, Rooney, CM, Spencer, DM, Straathof, KC, Vanin, EF, Yotnda, P   +8 more
core   +1 more source

Seeing Invisible Oligomers: Rethinking α‐Synuclein Pathology Through Proximity Ligation Assay

Movement Disorders, EarlyView.
Abstract Parkinson's disease (PD) and multiple system atrophy are defined by α‐synuclein (αSYN)‐positive inclusions – Lewy bodies (LBs) and glial cytoplasmic inclusions – yet mounting evidence indicates that these inclusions represent only a fraction of disease‐relevant pathology.
Hiroaki Sekiya, Nanna Møller Jensen, Dennis W. Dickson, Poul Henning Jensen   +3 more
wiley   +1 more source

Human Genome Safe Harbor Sites: A Comprehensive Review of Criteria, Discovery, Features, and Applications. [PDF]

Cells
Ahmed A, Di Molfetta D, Iaconisi GN, Caponio A, Singh A, Bibi A, Dolce V, Palmieri L, Coppola V, Fiermonte G.   +9 more
europepmc   +1 more source

Synthetic serum markers enable noninvasive monitoring of gene expression in primate brains. [PDF]

Neuron
Lee S, Romac MD, Watanabe S, Chernov M, Li H, Raisley E, Rothenhoefer KM, Dahlquist Z, Szablowski JO, Costa VD.   +9 more
europepmc   +1 more source

Risk assessment of plant-to-bacterium transgene flow associated with novel small synthetic genome (minisynplastome) platforms for plastid genetic engineering. [PDF]

FEMS Microbiol Ecol
Vincent AG, Fuentes Quispe IA, Majdi M, Dice LT, Harbison SA, Lenaghan SC, DeBruyn JM, Occhialini A.   +7 more
europepmc   +1 more source

Current Status of Kidney Xenotransplantation in Basic Research

Organ Medicine, EarlyView.
Kidney xenotransplantation is a potential solution to the organ shortage for end‐stage kidney disease. This review systematically elaborates on the application advances of donor pig gene editing technologies, the molecular mechanisms and regulatory strategies of xenogeneic immune rejection, and the optimization approaches of immune compatibility ...
Yu Luo, Bingzhuo Liu, Weijie Lai
wiley   +1 more source

Highly efficient gene editing via targeted Cas9 insertion into chicken housekeeping gene. [PDF]

Poult Sci
Jung KM, Klein R, Mony SI, Chen PR, Lee K, Lee HJ.   +5 more
europepmc   +1 more source

Hemophilia A: An Ideal Disease for Prenatal Therapy

Prenatal Diagnosis, EarlyView.
ABSTRACT Hemophilia A (HA) is the most common inherited coagulation defect. Current state‐of‐the‐art treatment consists of frequent administration of prophylactic infusions of coagulation factor VIII (FVIII) protein or bispecific antibodies that replace the cofactor function of FVIIIa to maintain hemostasis. However, these treatments are far from ideal,
Christopher D. Porada, Anthony Atala, Graça Almeida‐Porada   +2 more
wiley   +1 more source