Disclosed herein are methods and compositions for inactivating mutant genes associated with LCA, using engineered nucleases comprising a DNA binding domain and a cleavage domain or cleavage half-domain in conditions promoting the cleavage of the mutant genes. Polynucleotides encoding nucleases, vectors comprising polynucleotides encoding nucleases, and cells comprising polynucleotides encoding nucleases and/or cells comprising nucleases are also provided.

The present application provides a Capillary number-based method of isolating circulating rare cells from a blood sample from a subject using filtration parameters determined based on the measurement of hemorheological parameters of the sample. The present application also provides a method for determining filtration parameters in a microfluidic elasto-filtration process for isolating circulating rare cells from a blood sample from a subject. The present application further provides a device for isolating circulating rare cells from a blood sample from a subject and a non-transitory computer storage medium for performing methods described in the present application.

Viral promoters and compositions and methods of use thereof are provided. Compositions include viruses with impaired ability to reactivate from latency, and pharmaceutical compositions and method of use thereof. The genome of the viruses include one or more mutations that reduce expression from one or more promoters that regulate expression of viral genes during reactivation from latency. The mutation(s) are typically in a region of the viral genome that includes (i) promoter elements of the iP1 promoter of human cytomegalovirus, or the sequence of another virus corresponding thereto (e.g., an iP1 promoter homolog); (ii) promoter elements of the iP2 promoter of human cytomegalovirus, or sequence of another virus corresponding thereto (e.g., an iP2 promoter homolog); or (iii) a combination thereof. In some embodiments the virus encodes one or more heterologous antigens. The viruses can be used as vaccines to induce prophylactic and therapeutic immune responses in subjects in need thereof.

Markers of acute myeloid leukemia stem cells (AMLSC) are identified. The markers are differentially expressed in comparison with normal counterpart cells, and are useful as diagnostic and therapeutic targets.

A microfluidic chip for circulating tumor cell separation, comprising a first shell layer, a second shell layer, and a filter membrane between the first shell layer and the second shell layer. A first channel is formed between the filter membrane and the first shell layer; a second channel is formed between the filter membrane and the second shell layer; the first shell layer is provided with m input interfaces and n output interfaces, wherein m is greater than or equal to 1 and n is greater than or equal to 1; the second shell layer is provided with x input interfaces and y output interfaces, wherein x is greater than or equal to 1 and y is greater than or equal to 1. The chip is used for circulating tumor cell separation to achieve high flux, high efficiency, and a simple method, and facilitate promotion.

The invention relates to a method for culturing a subpopulation of circulating epithelial tumour cells from a body fluid of a human or animal suffering from an epithelial tumour, wherein cells contained in the body fluid each containing at least one cell nucleus are separated from the body fluid and cultured over at least 24 hours in suspension, with formation of spheroids.

A new sensitive cell biomarker of solid tumors and viral infection is identified in blood. This biomarker can be used to determine presence of carcinomas, sarcomas, and viruses, rapid determination of treatment response, early detection of cancer, early detection of cancer recurrence, and may be used to determine therapy.

The present invention relates to modified haematopoietic stem cells, methods for preparing them, and their use in therapy; as well as methods and reagents for expanding haematopoietic stem cells (HSC) and methods for treating haematological disorders. A particular aspect relates to a method for treating a disease or condition characterised by elevated YTHDF2 expression comprising administering to a patient an effective amount of an YTHDF2 inhibitor. Certain aspects of the invention rely on YTFIDF2 protein level or function and use of YTFIDF2 inhibitors.

The present invention provides bioengineered extracellular matrix model derived from decellularized Wharton's jelly matrix (DWJM) and methods for making and using the same. After decellularization, the DWJM is homogenized, frozen, and lyophilized in a mold to form a molded scaffold having a substantially uniform pore size, pore distribution, and matrix component distribution, and can be trimmed and shaped to any desired size. The bioengineered DWJM is able to maintain the stem cell qualities of cultured cells, which is useful in screening chemotherapy drugs that target cancers, especially cancer stem cell populations. The bioengineered DWJM possesses matrix components similar to the bone hematopoietic niche and is useful in expanding and maintaining hematopoietic stem cells as well as promoting bone regeneration and repair.

Some embodiments of the methods and compositions provided herein relate to the preparation surfactant protein-D (SP-D). Some embodiments include the expression of human SP-D in certain cell lines, and the purification of human SP-D from such cell lines. Some embodiments include the preparation of certain oligomeric forms of human SP-D.