A method for reducing heterogeneity of a virus preparation may include generating virus ions from the virus preparation, repeatedly increasing at least one of a temperature and an incubation period at the increased temperature of at least one of the virus preparation and the generated virus ions, measuring mass-to-charge ratios and charge magnitudes of at least some of the generated virus ions at each increase of the at least one of the temperature and the incubation period, determining a mass spectrum at each increase of the at least one of the temperature and the incubation period based on values of the respective mass-to-charge ratios and charge magnitudes, and determining, based on the mass spectrums, optimum ones of the temperature and the incubation period which together minimize, or at least reduce, a heterogeneity of the virus preparation without aggregation of virus capsids in the virus preparation.

The present disclosure relates to a method for constructing a producer cell and the producer cell obtained by the method, wherein the producer cell is for producing a retroviral vector carrying a nucleic acid fragment of interest.

The present disclosure relates to a method for constructing a producer cell and the producer cell obtained by the method, wherein the producer cell is for producing a retroviral vector carrying a nucleic acid fragment of interest.

The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.

The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.

Sized-based detection techniques for detection of bio-nanoparticles are described. Detection nanoparticles and methods of forming the detection nanoparticles that can be utilized in the techniques are described. Detection nanoparticles can include modified bacteriophage that express a linking agent for a specific binding agent. Detection nanoparticles can bind a functional bio-nanoparticle with high specificity through specific binding of one or more entities unique to the functional bio-nanoparticle of interest. A detection nanoparticle can target an entity of a bio-nanoparticle that is relevant to its function, and as such, the methods can provide improvements in detection of complete and functional bio-nanoparticles. Size-based detection regimes can include particle displacement measurement techniques based upon Brownian motion.

Disclosed herein are engineered AAV VP capsid polypeptides with the ability to assemble into virus particles and having improved tissue tropism to, for example, CNS tissues. The capsids are engineered using the high throughput discovery system described herein. In certain embodiments, provided herein are recombinant adeno-associated virus (AAV) VP capsid polypeptides having at least one mutation in a residue corresponding to residue 581 to residue 589 in SEQ ID NO: 1.

Disclosed herein are engineered AAV VP capsid polypeptides with the ability to assemble into virus particles and having improved tissue tropism to, for example, CNS tissues. The capsids are engineered using the high throughput discovery system described herein. In certain embodiments, provided herein are recombinant adeno-associated virus (AAV) VP capsid polypeptides having at least one mutation in a residue corresponding to residue 581 to residue 589 in SEQ ID NO: 1.

The present disclosure provides compositions and methods of restoring or enhancing visual function in an individual by administering to the individual a pharmaceutical composition comprising a recombinant adeno-associated viral (rAAV) vector having a polynucleotide sequence that encodes a medium wavelength cone opsin (MW-opsin). The MW-opsin is expressed in a retinal cell in the individual, thereby restoring or enhancing visual function.

The present disclosure provides compositions and methods of restoring or enhancing visual function in an individual by administering to the individual a pharmaceutical composition comprising a recombinant adeno-associated viral (rAAV) vector having a polynucleotide sequence that encodes a medium wavelength cone opsin (MW-opsin). The MW-opsin is expressed in a retinal cell in the individual, thereby restoring or enhancing visual function.